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Investment
Opportunities in
Pediatric Health
September 2016
Leading Change for Better Pediatric Care
Leading Pediatric Academic Medical Center
Patient diversity
African-American 6%
Asian 6%
Latino 62%
Caucasian 25%
Native American 0.06%
Other 1%
Capacity
357 active beds
5,500 employees
802 medical staff
Annual Statistics
16,000
inpatients admissions
350,000
outpatient visits
>110,000
individual patients
78,000
Emergency
Department visits
2,300 patients transported by
helicopter, learjet and ambulance
U.S. News & World report
ranked No.1
Children’s Hospital in California
Interdisciplinary Centers in Areas Critical to Pediatric Health
Center for Cancer and Blood Diseases
One of the nation’s largest pediatric hematology-
oncology programs. Includes the only federally
funded basic and translational research programs
studying sickle cell disease on the west coast. Large,
integrated clinical trials program(167 active studies).
Home to 3 international phase I clinical trial
consortia. 5,200 unique patients.
Center for Fetal and Neonatal Medicine
Treats the most critically ill preemies, newborns and
infants in Southern California.
Center for Endocrinology, Diabetes and
Metabolism
Provides care to nearly 2,000 children with diabetes
and more than 5,000 children with other endocrine
disorders.
Children’s Orthopaedic Center
One of the nation’s most comprehensive
programs seeing 23,500 patients and
performing 1,880 orthopaedic surgeries
annually.
Vision Center
The largest pediatric ophthalmology
program in the U.S.
Cardiac Center
One of the busiest cardiac centers in the
country, and serves as a global patient
reference center.
Neurosciences Center
Provides a comprehensive continuum of
care bringing together services from
neurology, neurosurgery, psychiatry,
neuro-psychology, neuro-radiology, physical
therapy, occupational therapy, dietetics and
social work
Complete range of pediatric specialties in 5
departments and 32 divisions all devoted to children
The Saban Research Institute
The Saban Research Institute at Children’s
Hospital Los Angeles comprises basic,
translational and clinical research and is one
of the few freestanding pediatric hospitals in
the country where scientific inquiry is
combined with clinical care devoted
exclusively to children.
Among U.S. children’s hospitals, ranks
eighth for NIH funding
• $32.7 million in NIH funding (FY 2015)
• $74.8 million total research funding (FY
2015)
The Institute includes 198,000 square feet of
wet and dry lab space
• Core facilities fully equipped with state of
the art instrumentation
• Cutting edge biomedical informatics and
imaging technologies.
Home to leading international pediatric
cancer consortia: New Approaches to
Neuroblastoma Therapy (NANT),
Therapeutic Advances in Childhood
Leukemia/Lymphoma (TACL), Pediatric
Blood and Marrow Transplant Consortium
(PBMTC)
• 200 current clinical trials
• 1,625 square foot Clinical Trials Unit
The Center for Innovation raises standards in pediatric care through the
development, commercialization and implementation of novel products and
services in partnership with industry, venture capitalists, investors,
entrepreneurs and philanthropists.
The Center for Innovation operates the Consortium for Technology and
Innovation in Pediatrics (CTIP) a pediatric health accelerator serving
innovators and start-ups nationwide and funded in part by the US Food and
Drug Administration.
We evaluate over 80 technologies each year that seek to significantly
improve pediatric standards of care while offering broader market
opportunities.
The Center for Innovation
OUR PROCESS
We have a deep understanding of
and access to pediatric markets
Pediatric clinical needs
Requirements for translating
medical technologies to the
bedside
Regulatory strategies needed to
bring pediatric technologies to
market
Reimbursement strategies
Drivers of clinical adoption and
appropriate commercial strategies
Unmet need
validation
Business
model/case
Product
development
Regulatory
approval
Clinical
acceptance
Reimbursement
and
implementation
OUR ADVANTAGES
We have access to unique resources to
support development & commercialization
A clinical advisory network in all
areas of pediatric specialty with
KOLs and domain area experts
Clinical and translational research
collaborators
Hospital administrators and leaders
7 FDA-funded sites accelerating
the commercialization of pediatric
medical devices
150+ children’s hospitals
participating in the International
Society for Pediatric Innovation
(iSPI)
Avexegen
A preclinical stage company developing Neuregulin-4 (NRG-4), a first-in-class oral therapy
consisting of a naturally occurring peptide that functions to promote gut mucosal healing in
patients suffering from Inflammatory Bowel Disease (IBD), as well as the orphan neonate
condition necrotizing enterocolitis (NEC). Given its novel mechanism of action (MOA),
NRG-4 is well positioned to make significant inroads into the IBD and NEC market segments,
which combined are projected to exceed $10 billion in total market size by 2021.
Nanovalent
A preclinical stage oncology company developing a targeted drug delivery platform that uses
polymerized liposomal nanoparticles that are highly robust, customizable, stable, cheap, and
easy to manufacture. Nanovalent will pursue orphan designation for expedited commercialization
of its first therapeutic candidate in Ewing Sarcoma, a rare pediatric cancer.
CerRx
A clinical stage oncology company seeking to improve the lives of cancer patients by
bringing to the marketplace unique therapeutics that target the ceramide pathway. CerRx is
developing a portfolio of lead compounds including an oral formulation of fenretinide to treat
pediatric cancers, including neuroblastoma.
Therapeutics
TheraPEDS
A preclinical stage drug delivery company developing proprietary mucoadhesive oral drug
formulations combined with taste masking chemistry to reformulate existing drugs for
improved compliance and dosing in pediatric (and elderly) patients. The pediatric medicines
market is projected to reach over $100B by 2019.
Hillhurst Biopharmaceuticals
A preclinical stage company developing HBI-002, an oral therapeutic targeting the
cytoprotective Heme Oxygenase metabolic pathway for patients suffering from conditions
associated with inflammation and apoptosis, such as sickle cell disease, transplantation,
and cerebral injury. This therapeutic enables the use of a gasotransmitter for which seven
completed Phase 1 and 2 clinical studies have demonstrated safety and tolerability, albeit with
other delivery modalities that are associated with toxicity and other issues. These indications
combined represent a greater than $6.8B market opportunity.
Therapeutics
Antibody-Dependent NK Exosome Therapeutic for Neuroblastoma, Leukemia and Other Cancers*
Antibody-Dependent Exosome Cytotoxicity (ADEC) represents a completely new approach to cancer
immunotherapy with applicability to a wide array of malignancies in children and adults. It’s advantages include
an allogeneic source, expanded from readily available PBMCs, a distinct mechanism of action from cytotoxic
chemotherapies and targeted therapies, synergy with antibody therapy and their small size to penetrate
sanctuary sites. *Available for licensing from CHLA
AM80, An oral therapy to reduce incidence of infections and related mortality in patients with
chemotherapy-induced neutropenia when used in combination with GCSF*
For 20 years, G-CSF (Neupogen®, filgrastim) daily injection has been an effective treatment for increasing
numbers of neutrophils and white blood count. However, despite this therapy, chemotherapy induced
neutropenia continues to be a problem with infections, febrile neutropenia and associated deaths.
Am80 is an oral therapy that increases the bactericidal activity of neutrophils. Increased bactericidal activity
reduces the risk of infection. Patients have a therapy that reduces their risk of infection and associated
mortality while on chemotherapy. *Available for licensing from CHLA
Antibody Blockade of Integrin Alpha6 for the Treatment of Relapse Leukemia and Other Cancers*
Current therapies address tumor shrinkage, but not the tumor microenvironment in metastatic disease (bone
marrow). Relapse of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) remain a
significant problem with a high rate of bone marrow metastasis. Invasive breast cancer remains a significant
problem (200,000 new cases diagnosed per year) in the US with a 20% mortality (relapse) rate. Bone marrow
metastasis remains a problem for all 3.
P5G10 – A first in class blocking antibody to integrin a6 prevents tumor cell adhesion to bone marrow stroma.
Reduced adhesion to bone marrow sensitizes tumor cells to chemotherapy. Patients have a therapy that
reduces their risk of relapse disease and mortality associated with metastatic disease. *Available for licensing
from CHLA
Therapeutics
Tourmaline Medical
A device company commercializing the STARFISH Luminal Expansion System (LXS), an innovative, patented device
solution for short bowel syndrome (SBS). SBS is a devastating condition with no current cure that afflicts both
pediatrics and adults. Currently, patient care and associated drugs to facilitate this care can impart costs of over $1M
per year per patient. Unique regulatory pathways will allow Tourmaline to gain rapid and relatively low cost market
access with a unique value proposition for the payor community. Patient care centers are highly concentrated allowing
for an efficient commercial model that will drive rapid revenue growth and break-even. The STARFISH LXS System
has been proven in small and large animals and published in over a dozen peer reviewed publications to date. The
total addressable market for this device is $3B.
Epipace*
An injectable micropacemaker for fetuses, and for children and adults, to control abnormal heart rhythms. Epipace is
developing an alternative approach to the new leadless pacemakers by implanting its micropacemaker on the exterior
of the heart, or epicardially. This safer minimally-invasive approach enables for the first time, the pacing of fetuses, and
offers significant benefits for children and adults. The addressable market for replacing epicardial systems and fetal
pacing is $800M. The total pacemaker market is close to $10B.
*Available for licensing from CHLA
LifeFlow by 410 Medical, A Rapid Infusion System
Pediatric sepsis is responsible for over 75,000 hospitalizations, 10,000 deaths, and $4.8 billion in cost in the US each
year. Multiple studies have shown that implementation of ACCM/PALS guidelines early in the course of sepsis,
particularly antibiotics and aggressive fluid resuscitation, saves lives. Unfortunately, fluid delivery guidelines are rarely
achieved in clinical practice. Numerous studies, and the experience of the Children’s Hospital Associated sepsis
collaborative, have shown that barriers to fluid delivery remain one of the most common reasons for ineffective
resuscitation in pediatric sepsis.The LifeFlow™ rapid infuser is a single-use, hand-operated, disposable device that
allows fast and efficient fluid delivery to critically ill patients with shock and sepsis. Designed to help overcome
common barriers to fluid resuscitation, the device is simple and intuitive, minimizes provider fatigue, and frees
providers to address other patient care issues simultaneously. This solution is particularly useful in pre-hospital and
emergency department settings where speed is important and personnel resources may be limited, and is also well
suited for inpatient, critical care, and operating room environments.
Therapeutic Devices
NeoLight
Jaundice is one of the most common and deadliest afflictions facing babies. It occurs in 6 out of 10 newborn
babies globally and affects over 2.4 million infants in the US alone. Jaundice phototherapy devices are a sub-set
of the neonatal equipment market, which is projected to reach $4B by 2019. NeoLight has developed an innovative
light-channeling technique which produces high-intensity light that will measurably reduce treatment time and
eliminate complications associated with current phototherapy equipment on the market. This technology can
potentially reduce treatment time by 40% thus saving the hospital $8000 per procedure.
Hypothermia Devices
Therapeutic hypothermia has been used as a treatment of choice for the management of inflammatory responses
associated with soft tissue injuries and spinal cord injury, to ameliorate the effects of traumatic brain injuries
and strokes, to improve neurologic outcomes after cardiac arrest and neonatal hypoxic-ischemic
encephalopathy. However, existing cooling devices are not designed to address the requirements of stability,
transportability and efficiency for treatment. Hypothermia Devices has developed flexible and ergonomic
cooling/heating pads to be placed in contact with the skin and can be electronically controlled in order to produce
rapid and efficient temperature regulation. The total addressable global market for this technology is $3.9B.
Polyvascular, A Stent Mounted Heart Valve
Congenital heart defects are the most common of all birth defects and remain amongst the leading causes of infant
mortality in the developed world. Multiple types of congenital heart defects require valve replacement surgery.
Pulmonary valves are usually replaced using cadaveric homograft valves, since mechanical valves have increased
risk of thrombosis in the pulmonary position. Homograft pulmonary valves are not commonly available, particularly
in sizes suitable for use in children. Moreover, these valves do not grow with the patient, becoming too small as the
child grows. All these factors result in the need for repeated valve replacement surgeries. Therefore, there is an
urgent need of solutions to limit the frequency of repeated valve replacement surgeries for these children.
Polyvascular is developing an innovative stent mounted heart valve that does not contain any biologic tissue, limiting
any immune sensitization and that can be delivered via cardiac catheterization, eliminating the need for open heart
surgery. This heart valve is also expandable over time and will follow the child's growth, eliminating the need for
multiple surgeries.
Therapeutic Devices
TESI, A point of care cell processing device for treating small bowel syndrome*
Necrotizing enterocolitis (NEC) is a gastrointestinal disease that affects premature infants. The current
standard of care requires surgical removal of necrotized tissue, which requires life-long management of
nutritional insufficiencies (transplant and/or arterial line feeding) associated with short bowel syndrome.
TESI is a device-based kit for processing salvaged small intestinal tissue during surgery to create bowel
segments for implantation. A subset of cells is seeded on a biodegradable scaffold, which is implanted into
the patient resulting in autologous tissue-engineered intestines. A proof of concept has been obtained in
non-clinical models and pediatric scalability is being pursued in non-clinical studies. *Available for licensing
from CHLA
Lully Sleep
According to the National Sleep Foundation, 36 Million children experience sleep disorders on a weekly
basis. Night terrors are one of the leading causes of children not sleeping well at young ages. It is
estimated that nearly 25% of children age 2-12 will experience at least one night terror. The Lully Sleep
Guardian is designed to bring this clinically validated, effective technique to the hands of parents. Based
on a child's specific sleep patterns the Lully system uses vibrations, prior to a transition into unhealthy
deep sleep. The Sleep Guardian is based on a tried-and-true technique called scheduled awakenings.
The Sleep Guardian's once-a-night vibrations partially wake your child, bringing him or her out of the night
terror causing sleep patterns. Because children have high sleep pressure, they almost immediately return
to deep sleep. This interrupts the unhealthy sleep cycle and prevents night terrors. Clinical studies have
shown that this method has reduced 90% of night terrors.
Therapeutic Devices
Vaximmune
A device company focused on preventing the transmission of the most common infectious agents from
mothers to their babies during labor, Group B Strep (GBS), Ureaplasma (UP) and E. Coli by developing
a proprietary, disposable culture device that allows for the collection, stabilization and concentration of the
relevant sample enabling the use of a rapid diagnostic to read out the test result. The kit contains a sample
collection swab, a disposable culture device and a lateral flow assay. The sample can be collected by the
patient or the physician and the test can be performed by the physician or the laboratory. This product
supports a $700MM market opportunity in the US and a combined opportunity of $2.8BB in the developed
world.
MIND Dx
Neuropsychiatric disorders are devastating illnesses where approximately one-fourth of adults have a
mental illness and nearly half will develop one mental illness during their lifetime. The economic cost of care
for mental illnesses is roughly $100 billion in year 2003 in the US and $193 billion are lost in the in earnings
and wages. The gold standard for diagnoses is clinical interviews and history, resulting in misdiagnoses as
high as 50% at initial presentation and time to proper diagnosis can take years. MindDx has developed
machine-learning based diagnostic algorithms from MR images of the brain to diagnose neuropsychiatric
disorders with high sensitivity and specificity. The market opportunity for ADHD is $800M and $40B for
ADHD, bipolar, schizophrenia, depression and other psychiatric disorders combined.
Deton Corp
Deton is a company committed to eliminating the need for collection of sputum (the phlegm that resides deep
in the airway) as a sample for the diagnosis of lower respiratory tract infections. Sputum sampling has a
50% failure rate resulting from the inability of the patient to produce a sample or sample contamination from
the mouth. For these patients, results cannot be provided, leading to empirical treatment which, in turn, leads
to the proliferation of antibiotic resistant bacteria. Failure rates are highest for pediatric and geriatric patients.
A cough sample is a simple and effective replacement for sputum. There are approximately 10M sputum
samples requested annually in the developed world, supporting a $1B market cap.
SOFTWARE
Diagnostic Devices
CrestaBio
There are approximately 1,200,000 people living with or in remission from blood cancer in the US, with an estimated
150,000 new cases diagnosed annually. Minimal residual disease (MRD), defined as the number of cancerous cells
found in the blood or bone marrow during or after treatment, is used to determine if a patient is free of cancer. MRD is
used to monitor the effectiveness of therapy, determine when a patient is in remission and for early detection of
relapse. When MRD is undetected and therefore untreated, it is the primary cause of relapse. Since the limit of
detection of the diagnostic technology defines when a patient is free of cancer, improving the limit of detection directly
results in an improvement in overall survival. Today flow cytometry has a 1 in 10,000 limit of detection and PCR can
detect 1 in 100,000 for some subsets of leukemia. CrestaBio has developed a technology that combines the benefits
of both methods into one test, while improving the sensitivity by three orders of magnitude. The technology allows for
the rapid and cost-effective detection followed by capture and genetic characterization of 1 in 100,000,000 cells.
AZA Technology, A Portable Ammonemia Detector (PAD)
Hyperammonemia is a metabolic condition characterized by elevated levels of ammonia in the blood. Increased entry
of ammonia to the brain is a primary cause of neurologic disorders, such as congenital deficiencies of urea cycle
enzymes, hepatic encephalopathies, Reye syndrome, several other metabolic disorders, and some toxic
encephalopathies. Early diagnosis and improved monitoring are important because hyperammonemia can be treated
by effective medical interventions. In children, 400 infants are born annually in the U.S. with mutations in one of 44
genes that affect several metabolic pathways, and 400,000 infants who are born before 37 weeks of gestation are at
risk for temporarily compromised urea cycles due to immature liver function. The current standard of practice requires
at least 3 ml of blood that must be transported to a central laboratory for immediate processing. The child must endure
intravenous puncture for blood near a central laboratory. Aza Technology, Inc has developed an innovative portable
device that measures ammonia levels in blood obtained by finger or heel stick. This Portable Ammonemia Detector
(PAD) uses a novel technology that adapts an inexpensive commercially available ammonia detector for use with
blood. PAD measures blood ammonia within seconds from 0.01 ml (10 µL) of blood obtained by a simple finger or heel
stick. PAD will allow screening for early diagnosis and thus save lives and protect cognitive function in many children.
In addition, subsequent management of these children will improve when PAD is deployed to the bedside and
outpatient clinics.
Diagnostic Devices
Hangar Hustle, A Game-Based Cognitive Assessment Tool
Attention deficits affect millions of people in the US; the percent of children 3-17 years of age with ADHD is 9.5%
and the prevalence of ADHD in the U.S. adult population is approximately 4.4%. Beyond the personal costs to
relationships, education, employment, is the economic cost to the nation in accidents, lost productivity, and
additional healthcare services. Expeditious assessment means earlier access to treatment that will reduce the
personal and economic impact of cognitive impairments. Blue Marble has developed an innovative game-based
assessment tool designed to measure various forms of attention, working memory and executive functions in the
course of video game play. This tool is intended to replace current paper-based or computerized assessment that
are cumbersome, time consuming and non-engaging for the patient. Using this new technology will reduce the
per-patient cost for assessment and will therefore stretch resources farther to serve more people.
Medulloblastoma Diagnostic Assay*
Medulloblastoma is the most common malignant pediatric brain tumor. Approximately 30% of patients remain
incurable and current radiation therapy containing treatment protocols cause significant adverse long-term
neurocognitive effects and endocrine dysfunction. In order to increase survival rates and reduce adverse
sequelae, there is a need of early detection methods and refined patient stratification. This 31-gene diagnostic
assay allows accurate identification of medulloblastoma subgroups and examines the expression of inflammation-
related genes with respect to each subgroup. Treatment for medulloblastoma is currently conducted with a multi-
modality approach (surgery, radiation therapy and chemotherapy) and the modality is heavily dependent on
tumor staging and risk group categorization. The molecular classification of medulloblastoma using this platform
will greatly improve current clinical risk stratification and therefore help with choosing the more efficient treatment
for the patient. *Available for licensing from CHLA
Neuroblastoma Prognostic Assay*
Neuroblastoma, an embryonal tumor of the peripheral sympathetic nervous system, is one of the most common
solid tumors in children, with approximately 45% of patients presenting with metastatic disease at diagnosis. Only
45% of those with high-risk metastatic neuroblastoma are cured with current treatments that include intensive
chemotherapy and immunotherapy. Having a new means to identify individuals at extremely high-risk for disease
progression and to assess response to therapy would be valuable for disease management.This 14-gene
prognostic assay identifies high and ultra-high risk groups and a highly sensitive and specific 5-gene response
assay to quantify circulating and bone marrow tumor cells in patients with neuroblastoma. These tests represent
an integrated disease management platform that may be particularly useful in adapting and evaluating
treatments, and in predicting outcome in children with neuroblastoma. A novel method for recovering high quality
RNA from viable fresh and frozen specimens was established for use in these tests. This platform, with
modifications, may also be useful in adults with cancer. *Available for licensing from CHLA
SOFTWARE
Diagnostic Devices
Next Generation Cystic Fibrosis Diagnostic Tool*
Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An
estimated 30,000 children and adults in the United States (70,000 worldwide) have CF. CF is caused by mutations in a
gene that produces a protein, called CFTR. The CFTR protein controls the flow of salt and water in and out of the cells
of organs like the lungs and pancreas. CF is usually diagnosed by conducting a QPIT sweat test, which measures the
amount of chloride in a person’s sweat. However, this test is only valid for patient with severe disease and does not
take into account the spectrum of pathologies associated with variable degrees of CFTR dysfunction. This is
particularly the case with adolescents, adults, and asymptomatic infants identified as being at risk for CF and
presenting with milder symptoms of the disease. This new method is based on beta adrenergically induced sweat
secretion. The rate of b-adrenergically stimulated sweating is used of diagnose CF disease and reflect the probability
of underlying genotypes. B-adrenergic sweat secretion rates are assessed with an evaporimeter to provide an
accurate, reliable and practical real-time assay over a range of CFTR functional activity in vivo. This new test will be a
very valuable asset to diagnose CF disease and a possible biomarker for CF drug efficacy testing. *Available for
licensing from CHLA
Diagnostic Devices
Prospiria, an optoaccountic guidance system for endotracheal tube placement
Over 25 million endotracheal tubes (ETs) are placed in patients each year. However, there is not an expedient
solution to identify the 20 to 50 percent of ETs that are initially malpositioned, or that move during intubation.
Incorrectly positioned ETs pose serious risk to patients, especially infants.
Prospiria has developed a patent-pending technology that uses optoacoustics (ultrasound signals generated by
pulsed laser light) to rapidly, precisely, and noninvasively identify the position of endotracheal tubes that have been
placed within the trachea. This technology will increase effectiveness and reduce complications by allowing
clinicians to quickly confirm and monitor both initial and subsequent ET placement.
Inceptio Medical Technologies
An estimated, 2.5 million peripherally inserted central catheters (PICC) line placements are performed in the U.S.
annually. Although a common procedure, PICC placements are particularly difficult in neonates because their
arteries have such small diameters and low pressure for palpitation. Complications that can arise from
misplacement include catheter induced perforation, thrombosis, and catheter induced arrhythmia. Inceptio has
developed a novel technology for precisely locating the distal tip of a cardiac catheter. The system provides
clinicians with real-time visualization during the procedure which greatly increasing the accuracy of placement and
reduces complications. The U.S. PICC market is project to reach over $583M by 2017.
Lifebubble, an Umbilical Catheter Protective Device
Critically ill neonates in the neonatal intensive care unit (NICU) receive umbilical catheters (UCs) because the
umbilical vein and artery in the umbilical cord are an easy and painless way to access the central circulation system.
Many of these babies are premature and have immature immune systems, leaving them susceptible to bloodstream
infections that are attributable to the presence of the umbilical catheter. The rate of UC central-line associated
bloodstream infections (CLABSIs) in neonates is at least five times greater than CLABSIs in the adult population.
Studies estimate that 5-15% of neonates with UCs develop CLABSIs, with rates highest for infants <1,250 grams.
For adult central lines, an occlusive bandage can be applied to the site of catheter insertion that is relatively flat to
the patient’s skin to keep it clean and dry. This standard cannot be applied to the UC that exits the vessel
perpendicular to the skin through a piece of tissue (umbilical stump) that has a unique shape and that must dry out
over the course of a few weeks. LIFEbubble is a innovative device that is tailored to the baby’s unique anatomy. It
replaces the non-sterile tape configuration to reduce the migration of bacteria along the skin, protect the umbilical
stump-catheter interface from accidental touch, and secure the UC to prevent entry of the non-clean catheter into
the bloodstream. LIFEbubble provides a streamlined solution to improve the current standard of care and save the
lives of vulnerable infants.
Positioning Devices
NonInvasix
NonInvasix is dedicated to the measurement and monitoring of venous oxygenation, delivering valuable
information regarding the adequacy of oxygen perfusion to the brain. A platform technology with a wide
clinical utility, the technology will first be applied to the monitoring of neonates in the NICU to predict
hypoxic ischemic encephalopathy (HIE). The inability to predict poor perfusion to the brain leads to
long term neurological deficit and can be avoided by monitoring brain venous oxygenation. HIE occurs at
a rate of 1 per 1,000 live births, costing $900,000 per patient per lifetime and is responsible for $3.6B in
annual economic burden.
GI Logic
Millions of adults, children and neonates struggle with digestive disorders, both acute and chronic.
Diagnosing digestive problems requires a hospital visit, use of expensive imaging equipment or expensive
invasive procedures. Current diagnostics monitor “one point in time episodes”, are expensive and not
always immediately available. GI Logic is commercializing “AbStats”, a non-invasive monitoring technology
that accurately measures intestinal activity “continuously” or a “snap shot”, providing an immediate
measurement of digestive health. AbStats can be used in a hospital setting, a clinic setting or a
residential environment. Disposable sensors prevent transfer of bacteria and web services interface
provides for easy storing of information in electronic health records.
BestDose*
Dosing errors make up 37% of all preventable medication errors at a cost of $8B to the healthcare system.
BestDose is developing an enterprise solution for individualized dosing, addressing a >$1B total market.
Its peer-reviewed software platform that combines critical patient data and important drug characteristics
to optimize dosing for any drug. Its solutions can be integrated into the EMR as part of the computerized
physician ordering process (CPOE) and improve prescriber and pharmacist efficiency by accurately
calculating dosages with a button click.
*Available for licensing from CHLA
SOFTWARE
SOFTWARE
Monitoring/CDS Devices
Respivice*
Over 800,000 US children are intubated and receive mechanical ventilation each year. Current
strategies to manage mechanical ventilators in children are inefficient, leading to prolonged intubation in
50% of patients and extubation failure in another 10% of patients. Respivce is a stand alone system that
provides quantitative measures to (1) optimize ventilator weaning, (2) easily identify the most common
reasons for extubation failure and (3) manage noninvasive ventilation after extubation. By avoiding these
complications Respivice can reduce healthcare costs by $6.75B in an addressable market of $600M.
*Available for licensing from CHLA
E-Vent, A Digital Ventilator Management Protocol*
Ventilation management is the accumulation of multiple decisions. This type of iterative decision making is
amenable to guidance using a medical treatment protocol. Medical treatment protocols facilitate
consistent, evidence-based decisions for equivalent patient states and digital protocols improve adherence
over paper protocols. E-Vent is an electronic decision support tool translated from an adult ventilator
protocol that improves management of pediatric and adult patients with acute hypoxemic respiratory
failure.
*Available for licensing from CHLA
4DX
A medical technology company established to commercialize four-dimensional lung imaging technology to
provide the spatial and functional information needed to optimize treatment for each patient with conditions
such as cystic fibrosis, asthma, emphysema and COPD. 4DX offers the resolution of CT with doses
comparable to X-Ray and functional insights of a pulmonary function test in a single platform. The software
has been successfully proven and patented and the company is now seeking to raise funds to scale their
SAAS business and commercialize custom designed and dedicated scanning hardware for increased
patients per hour of operation, reduced radiation dose and enhanced fidelity of scans. Enhanced scanning
rates will make each dedicated scanner a valuable additional source of revenue for the analysis service.
SOFTWARE
SOFTWARE
SOFTWARE
Monitoring/CDS Devices
Click Brain
The current standard of care using pediatric brain MRI for assessment of developmental disorders,
neurological trauma and diseases is time-consuming, qualitative, and requires visual assessment of a
multitude of 2D images. ClickBrain™ is a reimbursable, quantitative translational clinical decision support
software that compares patients’ brain MRI morphometry to healthy normal subjects. ClickBrain™ is
intended to significantly impact patient care by 1) increasing efficiency of interpretation and reporting, 2)
promoting uniformity of interpretation regardless of radiologist experience level, thus reducing risk of
misdiagnosis, and 3) enabling earlier diagnosis and quantitative longitudinal studies for treatment
monitoring.
TimeStamp
Cerebrospinal fluid (CSF) flow can de obstructed due to a variety of medical conditions such as infections,
meningitis, brain tumors, traumatic injuries etc. Because CSF is made continuously, medical conditions
that block its normal flow will result in an over-accumulation of CSF. The resulting pressure of the fluid
against brain tissue causes hydrocephalus. The incidence of pediatric hydrocephalous is about 1/1,000
births and early detection of CSF flow obstruction is a key component for proper treatment selection and
diminution of downstream effects. Current techniques generate images that lose tissue/CSF contrast over
the image capture process thereby complicating the radiologist's ability to identify CSF flow obstruction.
TimeStamp is a new MRI-based image acquisition software that allows MRI images to maintain high image
contrast for easier and faster interpretation by the radiologist.
SOFTWARE
SOFTWARE
Monitoring/CDS Devices
Qidza
CDC reports that 1 out 6 children have developmental or behavioral problems and 1 in 9 US births are
premature (Boyle et al.,2011), costing healthcare $30+ billion annually. Extensive research proves early
detection can improve outcomes and reduce costly interventions (Glascoe et al, 1997; WHO, 2007). Yet
52% of pediatricians don’t have time or expertise to screen for developmental issues (American Academy
of Pediatrics, 2014). To close the screening gap and offer parents peace of mind, Qidza is a mobile
platform that enables parents work seamlessly with their physicians to track their children’s developmental
milestones and improve their health and well‐being. Quizda has developed BabyNoggin, a preventative
online tool to monitor and track your baby’s brain development from birth to age two. Within minutes
you can screen your baby in the comfort of your home. The app offers an easy and fun way to interact with
your baby and track his or her development. Data gathered from BabyNogin will improve efficiency of well-
child visits to pediatrician and potentially improve clinical outcomes.
Neuromuscular Dynamics
It is well known that both sensory and motor systems contribute to the ability to maintain balance. Sensory
inputs are necessary to detect unstable conditions and motor contributions are vital to initiate timely and
appropriate responses to counteract these perturbations. The current clinical measures quantify the
functional domains of strength and multi-join coordination but they do not quantify the functional domain of
dexterity. This new strength-dexterity test device becomes unstable when compressed. Therefore, the
maximal level of sustained compression is informative of the maximal ability of the neuromuscular system
to stabilize unstable interactions. For the fingers, it is a metric of dexterous manipulation ability. For the
legs, it is a metric of leg dexterity and the ability of the leg to regulate dynamical interactions with the
ground.
SOFTWARE
Monitoring/CDS Devices
LifeSpeed
Lifespeed is a health and wellness platform that allows families, medical professionals, and caretakers to
safely store and share data. The tool provides deeper consumer engagement by offering a visually
compelling experience to enable users (starting with chronically ill and mums) to manage their health
profile efficiently. The consumer-centric platform/dashboard allows users to securely organize, archive and
share medical data such as x-rays, data from wearables, lab data, forms and appointments (analogous to
Pinterest + Dropbox). Glimpse allows users to invite doctors, caretakers or loved ones to view their data
securely. For providers, Lifespeed offers easy-to-use tools for digital content distribution, communication
and analysis of patient engagement. The integration of consumer populated data with existing medical
records creates an ecosystem that offers comprehensive collaboration between consumers and providers.
The platform facilitates a marketplace where stakeholders and third party developers bring apps to the
dashboard. The Lifespeed dashboard also provides a medium where healthcare organizations can
advertise to targeted communities through the consumer centered portal for health data management.
NICOlette
NICOlette’s first offering is a forthcoming parent-facing, HIPAA-compliant app called NicoBoard. It shares
NICU patients’ electronic health record (EHR) data in visually intuitive ways designed to allow parents to
quickly grasp the information. It extracts the EHR raw data and synthesizes it into a consumable format for
parents so that they can feel competent and confident in participating in their child’s journey to wellness. In
addition to parsing out easily digestible EHR data visualizations (such as color-coded graphs of a patient’s
oxygen saturation, carbon dioxide levels and apnea episode history, for example), NicoBoard will also
furnish parents with curated research relevant to newborn health. The idea is for parents to use the
research to make more informed healthcare decisions on behalf of their babies.
SOFTWARE
SOFTWARE
Patient Engagement
Thank You
Leading Change for Better Pediatric Care

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CHLA Pediatric Health Investment Opportunities_Sept 16

  • 1. Investment Opportunities in Pediatric Health September 2016 Leading Change for Better Pediatric Care
  • 2. Leading Pediatric Academic Medical Center Patient diversity African-American 6% Asian 6% Latino 62% Caucasian 25% Native American 0.06% Other 1% Capacity 357 active beds 5,500 employees 802 medical staff Annual Statistics 16,000 inpatients admissions 350,000 outpatient visits >110,000 individual patients 78,000 Emergency Department visits 2,300 patients transported by helicopter, learjet and ambulance U.S. News & World report ranked No.1 Children’s Hospital in California
  • 3. Interdisciplinary Centers in Areas Critical to Pediatric Health Center for Cancer and Blood Diseases One of the nation’s largest pediatric hematology- oncology programs. Includes the only federally funded basic and translational research programs studying sickle cell disease on the west coast. Large, integrated clinical trials program(167 active studies). Home to 3 international phase I clinical trial consortia. 5,200 unique patients. Center for Fetal and Neonatal Medicine Treats the most critically ill preemies, newborns and infants in Southern California. Center for Endocrinology, Diabetes and Metabolism Provides care to nearly 2,000 children with diabetes and more than 5,000 children with other endocrine disorders. Children’s Orthopaedic Center One of the nation’s most comprehensive programs seeing 23,500 patients and performing 1,880 orthopaedic surgeries annually. Vision Center The largest pediatric ophthalmology program in the U.S. Cardiac Center One of the busiest cardiac centers in the country, and serves as a global patient reference center. Neurosciences Center Provides a comprehensive continuum of care bringing together services from neurology, neurosurgery, psychiatry, neuro-psychology, neuro-radiology, physical therapy, occupational therapy, dietetics and social work Complete range of pediatric specialties in 5 departments and 32 divisions all devoted to children
  • 4. The Saban Research Institute The Saban Research Institute at Children’s Hospital Los Angeles comprises basic, translational and clinical research and is one of the few freestanding pediatric hospitals in the country where scientific inquiry is combined with clinical care devoted exclusively to children. Among U.S. children’s hospitals, ranks eighth for NIH funding • $32.7 million in NIH funding (FY 2015) • $74.8 million total research funding (FY 2015) The Institute includes 198,000 square feet of wet and dry lab space • Core facilities fully equipped with state of the art instrumentation • Cutting edge biomedical informatics and imaging technologies. Home to leading international pediatric cancer consortia: New Approaches to Neuroblastoma Therapy (NANT), Therapeutic Advances in Childhood Leukemia/Lymphoma (TACL), Pediatric Blood and Marrow Transplant Consortium (PBMTC) • 200 current clinical trials • 1,625 square foot Clinical Trials Unit
  • 5. The Center for Innovation raises standards in pediatric care through the development, commercialization and implementation of novel products and services in partnership with industry, venture capitalists, investors, entrepreneurs and philanthropists. The Center for Innovation operates the Consortium for Technology and Innovation in Pediatrics (CTIP) a pediatric health accelerator serving innovators and start-ups nationwide and funded in part by the US Food and Drug Administration. We evaluate over 80 technologies each year that seek to significantly improve pediatric standards of care while offering broader market opportunities. The Center for Innovation
  • 6. OUR PROCESS We have a deep understanding of and access to pediatric markets Pediatric clinical needs Requirements for translating medical technologies to the bedside Regulatory strategies needed to bring pediatric technologies to market Reimbursement strategies Drivers of clinical adoption and appropriate commercial strategies Unmet need validation Business model/case Product development Regulatory approval Clinical acceptance Reimbursement and implementation
  • 7. OUR ADVANTAGES We have access to unique resources to support development & commercialization A clinical advisory network in all areas of pediatric specialty with KOLs and domain area experts Clinical and translational research collaborators Hospital administrators and leaders 7 FDA-funded sites accelerating the commercialization of pediatric medical devices 150+ children’s hospitals participating in the International Society for Pediatric Innovation (iSPI)
  • 8. Avexegen A preclinical stage company developing Neuregulin-4 (NRG-4), a first-in-class oral therapy consisting of a naturally occurring peptide that functions to promote gut mucosal healing in patients suffering from Inflammatory Bowel Disease (IBD), as well as the orphan neonate condition necrotizing enterocolitis (NEC). Given its novel mechanism of action (MOA), NRG-4 is well positioned to make significant inroads into the IBD and NEC market segments, which combined are projected to exceed $10 billion in total market size by 2021. Nanovalent A preclinical stage oncology company developing a targeted drug delivery platform that uses polymerized liposomal nanoparticles that are highly robust, customizable, stable, cheap, and easy to manufacture. Nanovalent will pursue orphan designation for expedited commercialization of its first therapeutic candidate in Ewing Sarcoma, a rare pediatric cancer. CerRx A clinical stage oncology company seeking to improve the lives of cancer patients by bringing to the marketplace unique therapeutics that target the ceramide pathway. CerRx is developing a portfolio of lead compounds including an oral formulation of fenretinide to treat pediatric cancers, including neuroblastoma. Therapeutics
  • 9. TheraPEDS A preclinical stage drug delivery company developing proprietary mucoadhesive oral drug formulations combined with taste masking chemistry to reformulate existing drugs for improved compliance and dosing in pediatric (and elderly) patients. The pediatric medicines market is projected to reach over $100B by 2019. Hillhurst Biopharmaceuticals A preclinical stage company developing HBI-002, an oral therapeutic targeting the cytoprotective Heme Oxygenase metabolic pathway for patients suffering from conditions associated with inflammation and apoptosis, such as sickle cell disease, transplantation, and cerebral injury. This therapeutic enables the use of a gasotransmitter for which seven completed Phase 1 and 2 clinical studies have demonstrated safety and tolerability, albeit with other delivery modalities that are associated with toxicity and other issues. These indications combined represent a greater than $6.8B market opportunity. Therapeutics
  • 10. Antibody-Dependent NK Exosome Therapeutic for Neuroblastoma, Leukemia and Other Cancers* Antibody-Dependent Exosome Cytotoxicity (ADEC) represents a completely new approach to cancer immunotherapy with applicability to a wide array of malignancies in children and adults. It’s advantages include an allogeneic source, expanded from readily available PBMCs, a distinct mechanism of action from cytotoxic chemotherapies and targeted therapies, synergy with antibody therapy and their small size to penetrate sanctuary sites. *Available for licensing from CHLA AM80, An oral therapy to reduce incidence of infections and related mortality in patients with chemotherapy-induced neutropenia when used in combination with GCSF* For 20 years, G-CSF (Neupogen®, filgrastim) daily injection has been an effective treatment for increasing numbers of neutrophils and white blood count. However, despite this therapy, chemotherapy induced neutropenia continues to be a problem with infections, febrile neutropenia and associated deaths. Am80 is an oral therapy that increases the bactericidal activity of neutrophils. Increased bactericidal activity reduces the risk of infection. Patients have a therapy that reduces their risk of infection and associated mortality while on chemotherapy. *Available for licensing from CHLA Antibody Blockade of Integrin Alpha6 for the Treatment of Relapse Leukemia and Other Cancers* Current therapies address tumor shrinkage, but not the tumor microenvironment in metastatic disease (bone marrow). Relapse of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) remain a significant problem with a high rate of bone marrow metastasis. Invasive breast cancer remains a significant problem (200,000 new cases diagnosed per year) in the US with a 20% mortality (relapse) rate. Bone marrow metastasis remains a problem for all 3. P5G10 – A first in class blocking antibody to integrin a6 prevents tumor cell adhesion to bone marrow stroma. Reduced adhesion to bone marrow sensitizes tumor cells to chemotherapy. Patients have a therapy that reduces their risk of relapse disease and mortality associated with metastatic disease. *Available for licensing from CHLA Therapeutics
  • 11. Tourmaline Medical A device company commercializing the STARFISH Luminal Expansion System (LXS), an innovative, patented device solution for short bowel syndrome (SBS). SBS is a devastating condition with no current cure that afflicts both pediatrics and adults. Currently, patient care and associated drugs to facilitate this care can impart costs of over $1M per year per patient. Unique regulatory pathways will allow Tourmaline to gain rapid and relatively low cost market access with a unique value proposition for the payor community. Patient care centers are highly concentrated allowing for an efficient commercial model that will drive rapid revenue growth and break-even. The STARFISH LXS System has been proven in small and large animals and published in over a dozen peer reviewed publications to date. The total addressable market for this device is $3B. Epipace* An injectable micropacemaker for fetuses, and for children and adults, to control abnormal heart rhythms. Epipace is developing an alternative approach to the new leadless pacemakers by implanting its micropacemaker on the exterior of the heart, or epicardially. This safer minimally-invasive approach enables for the first time, the pacing of fetuses, and offers significant benefits for children and adults. The addressable market for replacing epicardial systems and fetal pacing is $800M. The total pacemaker market is close to $10B. *Available for licensing from CHLA LifeFlow by 410 Medical, A Rapid Infusion System Pediatric sepsis is responsible for over 75,000 hospitalizations, 10,000 deaths, and $4.8 billion in cost in the US each year. Multiple studies have shown that implementation of ACCM/PALS guidelines early in the course of sepsis, particularly antibiotics and aggressive fluid resuscitation, saves lives. Unfortunately, fluid delivery guidelines are rarely achieved in clinical practice. Numerous studies, and the experience of the Children’s Hospital Associated sepsis collaborative, have shown that barriers to fluid delivery remain one of the most common reasons for ineffective resuscitation in pediatric sepsis.The LifeFlow™ rapid infuser is a single-use, hand-operated, disposable device that allows fast and efficient fluid delivery to critically ill patients with shock and sepsis. Designed to help overcome common barriers to fluid resuscitation, the device is simple and intuitive, minimizes provider fatigue, and frees providers to address other patient care issues simultaneously. This solution is particularly useful in pre-hospital and emergency department settings where speed is important and personnel resources may be limited, and is also well suited for inpatient, critical care, and operating room environments. Therapeutic Devices
  • 12. NeoLight Jaundice is one of the most common and deadliest afflictions facing babies. It occurs in 6 out of 10 newborn babies globally and affects over 2.4 million infants in the US alone. Jaundice phototherapy devices are a sub-set of the neonatal equipment market, which is projected to reach $4B by 2019. NeoLight has developed an innovative light-channeling technique which produces high-intensity light that will measurably reduce treatment time and eliminate complications associated with current phototherapy equipment on the market. This technology can potentially reduce treatment time by 40% thus saving the hospital $8000 per procedure. Hypothermia Devices Therapeutic hypothermia has been used as a treatment of choice for the management of inflammatory responses associated with soft tissue injuries and spinal cord injury, to ameliorate the effects of traumatic brain injuries and strokes, to improve neurologic outcomes after cardiac arrest and neonatal hypoxic-ischemic encephalopathy. However, existing cooling devices are not designed to address the requirements of stability, transportability and efficiency for treatment. Hypothermia Devices has developed flexible and ergonomic cooling/heating pads to be placed in contact with the skin and can be electronically controlled in order to produce rapid and efficient temperature regulation. The total addressable global market for this technology is $3.9B. Polyvascular, A Stent Mounted Heart Valve Congenital heart defects are the most common of all birth defects and remain amongst the leading causes of infant mortality in the developed world. Multiple types of congenital heart defects require valve replacement surgery. Pulmonary valves are usually replaced using cadaveric homograft valves, since mechanical valves have increased risk of thrombosis in the pulmonary position. Homograft pulmonary valves are not commonly available, particularly in sizes suitable for use in children. Moreover, these valves do not grow with the patient, becoming too small as the child grows. All these factors result in the need for repeated valve replacement surgeries. Therefore, there is an urgent need of solutions to limit the frequency of repeated valve replacement surgeries for these children. Polyvascular is developing an innovative stent mounted heart valve that does not contain any biologic tissue, limiting any immune sensitization and that can be delivered via cardiac catheterization, eliminating the need for open heart surgery. This heart valve is also expandable over time and will follow the child's growth, eliminating the need for multiple surgeries. Therapeutic Devices
  • 13. TESI, A point of care cell processing device for treating small bowel syndrome* Necrotizing enterocolitis (NEC) is a gastrointestinal disease that affects premature infants. The current standard of care requires surgical removal of necrotized tissue, which requires life-long management of nutritional insufficiencies (transplant and/or arterial line feeding) associated with short bowel syndrome. TESI is a device-based kit for processing salvaged small intestinal tissue during surgery to create bowel segments for implantation. A subset of cells is seeded on a biodegradable scaffold, which is implanted into the patient resulting in autologous tissue-engineered intestines. A proof of concept has been obtained in non-clinical models and pediatric scalability is being pursued in non-clinical studies. *Available for licensing from CHLA Lully Sleep According to the National Sleep Foundation, 36 Million children experience sleep disorders on a weekly basis. Night terrors are one of the leading causes of children not sleeping well at young ages. It is estimated that nearly 25% of children age 2-12 will experience at least one night terror. The Lully Sleep Guardian is designed to bring this clinically validated, effective technique to the hands of parents. Based on a child's specific sleep patterns the Lully system uses vibrations, prior to a transition into unhealthy deep sleep. The Sleep Guardian is based on a tried-and-true technique called scheduled awakenings. The Sleep Guardian's once-a-night vibrations partially wake your child, bringing him or her out of the night terror causing sleep patterns. Because children have high sleep pressure, they almost immediately return to deep sleep. This interrupts the unhealthy sleep cycle and prevents night terrors. Clinical studies have shown that this method has reduced 90% of night terrors. Therapeutic Devices
  • 14. Vaximmune A device company focused on preventing the transmission of the most common infectious agents from mothers to their babies during labor, Group B Strep (GBS), Ureaplasma (UP) and E. Coli by developing a proprietary, disposable culture device that allows for the collection, stabilization and concentration of the relevant sample enabling the use of a rapid diagnostic to read out the test result. The kit contains a sample collection swab, a disposable culture device and a lateral flow assay. The sample can be collected by the patient or the physician and the test can be performed by the physician or the laboratory. This product supports a $700MM market opportunity in the US and a combined opportunity of $2.8BB in the developed world. MIND Dx Neuropsychiatric disorders are devastating illnesses where approximately one-fourth of adults have a mental illness and nearly half will develop one mental illness during their lifetime. The economic cost of care for mental illnesses is roughly $100 billion in year 2003 in the US and $193 billion are lost in the in earnings and wages. The gold standard for diagnoses is clinical interviews and history, resulting in misdiagnoses as high as 50% at initial presentation and time to proper diagnosis can take years. MindDx has developed machine-learning based diagnostic algorithms from MR images of the brain to diagnose neuropsychiatric disorders with high sensitivity and specificity. The market opportunity for ADHD is $800M and $40B for ADHD, bipolar, schizophrenia, depression and other psychiatric disorders combined. Deton Corp Deton is a company committed to eliminating the need for collection of sputum (the phlegm that resides deep in the airway) as a sample for the diagnosis of lower respiratory tract infections. Sputum sampling has a 50% failure rate resulting from the inability of the patient to produce a sample or sample contamination from the mouth. For these patients, results cannot be provided, leading to empirical treatment which, in turn, leads to the proliferation of antibiotic resistant bacteria. Failure rates are highest for pediatric and geriatric patients. A cough sample is a simple and effective replacement for sputum. There are approximately 10M sputum samples requested annually in the developed world, supporting a $1B market cap. SOFTWARE Diagnostic Devices
  • 15. CrestaBio There are approximately 1,200,000 people living with or in remission from blood cancer in the US, with an estimated 150,000 new cases diagnosed annually. Minimal residual disease (MRD), defined as the number of cancerous cells found in the blood or bone marrow during or after treatment, is used to determine if a patient is free of cancer. MRD is used to monitor the effectiveness of therapy, determine when a patient is in remission and for early detection of relapse. When MRD is undetected and therefore untreated, it is the primary cause of relapse. Since the limit of detection of the diagnostic technology defines when a patient is free of cancer, improving the limit of detection directly results in an improvement in overall survival. Today flow cytometry has a 1 in 10,000 limit of detection and PCR can detect 1 in 100,000 for some subsets of leukemia. CrestaBio has developed a technology that combines the benefits of both methods into one test, while improving the sensitivity by three orders of magnitude. The technology allows for the rapid and cost-effective detection followed by capture and genetic characterization of 1 in 100,000,000 cells. AZA Technology, A Portable Ammonemia Detector (PAD) Hyperammonemia is a metabolic condition characterized by elevated levels of ammonia in the blood. Increased entry of ammonia to the brain is a primary cause of neurologic disorders, such as congenital deficiencies of urea cycle enzymes, hepatic encephalopathies, Reye syndrome, several other metabolic disorders, and some toxic encephalopathies. Early diagnosis and improved monitoring are important because hyperammonemia can be treated by effective medical interventions. In children, 400 infants are born annually in the U.S. with mutations in one of 44 genes that affect several metabolic pathways, and 400,000 infants who are born before 37 weeks of gestation are at risk for temporarily compromised urea cycles due to immature liver function. The current standard of practice requires at least 3 ml of blood that must be transported to a central laboratory for immediate processing. The child must endure intravenous puncture for blood near a central laboratory. Aza Technology, Inc has developed an innovative portable device that measures ammonia levels in blood obtained by finger or heel stick. This Portable Ammonemia Detector (PAD) uses a novel technology that adapts an inexpensive commercially available ammonia detector for use with blood. PAD measures blood ammonia within seconds from 0.01 ml (10 µL) of blood obtained by a simple finger or heel stick. PAD will allow screening for early diagnosis and thus save lives and protect cognitive function in many children. In addition, subsequent management of these children will improve when PAD is deployed to the bedside and outpatient clinics. Diagnostic Devices
  • 16. Hangar Hustle, A Game-Based Cognitive Assessment Tool Attention deficits affect millions of people in the US; the percent of children 3-17 years of age with ADHD is 9.5% and the prevalence of ADHD in the U.S. adult population is approximately 4.4%. Beyond the personal costs to relationships, education, employment, is the economic cost to the nation in accidents, lost productivity, and additional healthcare services. Expeditious assessment means earlier access to treatment that will reduce the personal and economic impact of cognitive impairments. Blue Marble has developed an innovative game-based assessment tool designed to measure various forms of attention, working memory and executive functions in the course of video game play. This tool is intended to replace current paper-based or computerized assessment that are cumbersome, time consuming and non-engaging for the patient. Using this new technology will reduce the per-patient cost for assessment and will therefore stretch resources farther to serve more people. Medulloblastoma Diagnostic Assay* Medulloblastoma is the most common malignant pediatric brain tumor. Approximately 30% of patients remain incurable and current radiation therapy containing treatment protocols cause significant adverse long-term neurocognitive effects and endocrine dysfunction. In order to increase survival rates and reduce adverse sequelae, there is a need of early detection methods and refined patient stratification. This 31-gene diagnostic assay allows accurate identification of medulloblastoma subgroups and examines the expression of inflammation- related genes with respect to each subgroup. Treatment for medulloblastoma is currently conducted with a multi- modality approach (surgery, radiation therapy and chemotherapy) and the modality is heavily dependent on tumor staging and risk group categorization. The molecular classification of medulloblastoma using this platform will greatly improve current clinical risk stratification and therefore help with choosing the more efficient treatment for the patient. *Available for licensing from CHLA Neuroblastoma Prognostic Assay* Neuroblastoma, an embryonal tumor of the peripheral sympathetic nervous system, is one of the most common solid tumors in children, with approximately 45% of patients presenting with metastatic disease at diagnosis. Only 45% of those with high-risk metastatic neuroblastoma are cured with current treatments that include intensive chemotherapy and immunotherapy. Having a new means to identify individuals at extremely high-risk for disease progression and to assess response to therapy would be valuable for disease management.This 14-gene prognostic assay identifies high and ultra-high risk groups and a highly sensitive and specific 5-gene response assay to quantify circulating and bone marrow tumor cells in patients with neuroblastoma. These tests represent an integrated disease management platform that may be particularly useful in adapting and evaluating treatments, and in predicting outcome in children with neuroblastoma. A novel method for recovering high quality RNA from viable fresh and frozen specimens was established for use in these tests. This platform, with modifications, may also be useful in adults with cancer. *Available for licensing from CHLA SOFTWARE Diagnostic Devices
  • 17. Next Generation Cystic Fibrosis Diagnostic Tool* Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An estimated 30,000 children and adults in the United States (70,000 worldwide) have CF. CF is caused by mutations in a gene that produces a protein, called CFTR. The CFTR protein controls the flow of salt and water in and out of the cells of organs like the lungs and pancreas. CF is usually diagnosed by conducting a QPIT sweat test, which measures the amount of chloride in a person’s sweat. However, this test is only valid for patient with severe disease and does not take into account the spectrum of pathologies associated with variable degrees of CFTR dysfunction. This is particularly the case with adolescents, adults, and asymptomatic infants identified as being at risk for CF and presenting with milder symptoms of the disease. This new method is based on beta adrenergically induced sweat secretion. The rate of b-adrenergically stimulated sweating is used of diagnose CF disease and reflect the probability of underlying genotypes. B-adrenergic sweat secretion rates are assessed with an evaporimeter to provide an accurate, reliable and practical real-time assay over a range of CFTR functional activity in vivo. This new test will be a very valuable asset to diagnose CF disease and a possible biomarker for CF drug efficacy testing. *Available for licensing from CHLA Diagnostic Devices
  • 18. Prospiria, an optoaccountic guidance system for endotracheal tube placement Over 25 million endotracheal tubes (ETs) are placed in patients each year. However, there is not an expedient solution to identify the 20 to 50 percent of ETs that are initially malpositioned, or that move during intubation. Incorrectly positioned ETs pose serious risk to patients, especially infants. Prospiria has developed a patent-pending technology that uses optoacoustics (ultrasound signals generated by pulsed laser light) to rapidly, precisely, and noninvasively identify the position of endotracheal tubes that have been placed within the trachea. This technology will increase effectiveness and reduce complications by allowing clinicians to quickly confirm and monitor both initial and subsequent ET placement. Inceptio Medical Technologies An estimated, 2.5 million peripherally inserted central catheters (PICC) line placements are performed in the U.S. annually. Although a common procedure, PICC placements are particularly difficult in neonates because their arteries have such small diameters and low pressure for palpitation. Complications that can arise from misplacement include catheter induced perforation, thrombosis, and catheter induced arrhythmia. Inceptio has developed a novel technology for precisely locating the distal tip of a cardiac catheter. The system provides clinicians with real-time visualization during the procedure which greatly increasing the accuracy of placement and reduces complications. The U.S. PICC market is project to reach over $583M by 2017. Lifebubble, an Umbilical Catheter Protective Device Critically ill neonates in the neonatal intensive care unit (NICU) receive umbilical catheters (UCs) because the umbilical vein and artery in the umbilical cord are an easy and painless way to access the central circulation system. Many of these babies are premature and have immature immune systems, leaving them susceptible to bloodstream infections that are attributable to the presence of the umbilical catheter. The rate of UC central-line associated bloodstream infections (CLABSIs) in neonates is at least five times greater than CLABSIs in the adult population. Studies estimate that 5-15% of neonates with UCs develop CLABSIs, with rates highest for infants <1,250 grams. For adult central lines, an occlusive bandage can be applied to the site of catheter insertion that is relatively flat to the patient’s skin to keep it clean and dry. This standard cannot be applied to the UC that exits the vessel perpendicular to the skin through a piece of tissue (umbilical stump) that has a unique shape and that must dry out over the course of a few weeks. LIFEbubble is a innovative device that is tailored to the baby’s unique anatomy. It replaces the non-sterile tape configuration to reduce the migration of bacteria along the skin, protect the umbilical stump-catheter interface from accidental touch, and secure the UC to prevent entry of the non-clean catheter into the bloodstream. LIFEbubble provides a streamlined solution to improve the current standard of care and save the lives of vulnerable infants. Positioning Devices
  • 19. NonInvasix NonInvasix is dedicated to the measurement and monitoring of venous oxygenation, delivering valuable information regarding the adequacy of oxygen perfusion to the brain. A platform technology with a wide clinical utility, the technology will first be applied to the monitoring of neonates in the NICU to predict hypoxic ischemic encephalopathy (HIE). The inability to predict poor perfusion to the brain leads to long term neurological deficit and can be avoided by monitoring brain venous oxygenation. HIE occurs at a rate of 1 per 1,000 live births, costing $900,000 per patient per lifetime and is responsible for $3.6B in annual economic burden. GI Logic Millions of adults, children and neonates struggle with digestive disorders, both acute and chronic. Diagnosing digestive problems requires a hospital visit, use of expensive imaging equipment or expensive invasive procedures. Current diagnostics monitor “one point in time episodes”, are expensive and not always immediately available. GI Logic is commercializing “AbStats”, a non-invasive monitoring technology that accurately measures intestinal activity “continuously” or a “snap shot”, providing an immediate measurement of digestive health. AbStats can be used in a hospital setting, a clinic setting or a residential environment. Disposable sensors prevent transfer of bacteria and web services interface provides for easy storing of information in electronic health records. BestDose* Dosing errors make up 37% of all preventable medication errors at a cost of $8B to the healthcare system. BestDose is developing an enterprise solution for individualized dosing, addressing a >$1B total market. Its peer-reviewed software platform that combines critical patient data and important drug characteristics to optimize dosing for any drug. Its solutions can be integrated into the EMR as part of the computerized physician ordering process (CPOE) and improve prescriber and pharmacist efficiency by accurately calculating dosages with a button click. *Available for licensing from CHLA SOFTWARE SOFTWARE Monitoring/CDS Devices
  • 20. Respivice* Over 800,000 US children are intubated and receive mechanical ventilation each year. Current strategies to manage mechanical ventilators in children are inefficient, leading to prolonged intubation in 50% of patients and extubation failure in another 10% of patients. Respivce is a stand alone system that provides quantitative measures to (1) optimize ventilator weaning, (2) easily identify the most common reasons for extubation failure and (3) manage noninvasive ventilation after extubation. By avoiding these complications Respivice can reduce healthcare costs by $6.75B in an addressable market of $600M. *Available for licensing from CHLA E-Vent, A Digital Ventilator Management Protocol* Ventilation management is the accumulation of multiple decisions. This type of iterative decision making is amenable to guidance using a medical treatment protocol. Medical treatment protocols facilitate consistent, evidence-based decisions for equivalent patient states and digital protocols improve adherence over paper protocols. E-Vent is an electronic decision support tool translated from an adult ventilator protocol that improves management of pediatric and adult patients with acute hypoxemic respiratory failure. *Available for licensing from CHLA 4DX A medical technology company established to commercialize four-dimensional lung imaging technology to provide the spatial and functional information needed to optimize treatment for each patient with conditions such as cystic fibrosis, asthma, emphysema and COPD. 4DX offers the resolution of CT with doses comparable to X-Ray and functional insights of a pulmonary function test in a single platform. The software has been successfully proven and patented and the company is now seeking to raise funds to scale their SAAS business and commercialize custom designed and dedicated scanning hardware for increased patients per hour of operation, reduced radiation dose and enhanced fidelity of scans. Enhanced scanning rates will make each dedicated scanner a valuable additional source of revenue for the analysis service. SOFTWARE SOFTWARE SOFTWARE Monitoring/CDS Devices
  • 21. Click Brain The current standard of care using pediatric brain MRI for assessment of developmental disorders, neurological trauma and diseases is time-consuming, qualitative, and requires visual assessment of a multitude of 2D images. ClickBrain™ is a reimbursable, quantitative translational clinical decision support software that compares patients’ brain MRI morphometry to healthy normal subjects. ClickBrain™ is intended to significantly impact patient care by 1) increasing efficiency of interpretation and reporting, 2) promoting uniformity of interpretation regardless of radiologist experience level, thus reducing risk of misdiagnosis, and 3) enabling earlier diagnosis and quantitative longitudinal studies for treatment monitoring. TimeStamp Cerebrospinal fluid (CSF) flow can de obstructed due to a variety of medical conditions such as infections, meningitis, brain tumors, traumatic injuries etc. Because CSF is made continuously, medical conditions that block its normal flow will result in an over-accumulation of CSF. The resulting pressure of the fluid against brain tissue causes hydrocephalus. The incidence of pediatric hydrocephalous is about 1/1,000 births and early detection of CSF flow obstruction is a key component for proper treatment selection and diminution of downstream effects. Current techniques generate images that lose tissue/CSF contrast over the image capture process thereby complicating the radiologist's ability to identify CSF flow obstruction. TimeStamp is a new MRI-based image acquisition software that allows MRI images to maintain high image contrast for easier and faster interpretation by the radiologist. SOFTWARE SOFTWARE Monitoring/CDS Devices
  • 22. Qidza CDC reports that 1 out 6 children have developmental or behavioral problems and 1 in 9 US births are premature (Boyle et al.,2011), costing healthcare $30+ billion annually. Extensive research proves early detection can improve outcomes and reduce costly interventions (Glascoe et al, 1997; WHO, 2007). Yet 52% of pediatricians don’t have time or expertise to screen for developmental issues (American Academy of Pediatrics, 2014). To close the screening gap and offer parents peace of mind, Qidza is a mobile platform that enables parents work seamlessly with their physicians to track their children’s developmental milestones and improve their health and well‐being. Quizda has developed BabyNoggin, a preventative online tool to monitor and track your baby’s brain development from birth to age two. Within minutes you can screen your baby in the comfort of your home. The app offers an easy and fun way to interact with your baby and track his or her development. Data gathered from BabyNogin will improve efficiency of well- child visits to pediatrician and potentially improve clinical outcomes. Neuromuscular Dynamics It is well known that both sensory and motor systems contribute to the ability to maintain balance. Sensory inputs are necessary to detect unstable conditions and motor contributions are vital to initiate timely and appropriate responses to counteract these perturbations. The current clinical measures quantify the functional domains of strength and multi-join coordination but they do not quantify the functional domain of dexterity. This new strength-dexterity test device becomes unstable when compressed. Therefore, the maximal level of sustained compression is informative of the maximal ability of the neuromuscular system to stabilize unstable interactions. For the fingers, it is a metric of dexterous manipulation ability. For the legs, it is a metric of leg dexterity and the ability of the leg to regulate dynamical interactions with the ground. SOFTWARE Monitoring/CDS Devices
  • 23. LifeSpeed Lifespeed is a health and wellness platform that allows families, medical professionals, and caretakers to safely store and share data. The tool provides deeper consumer engagement by offering a visually compelling experience to enable users (starting with chronically ill and mums) to manage their health profile efficiently. The consumer-centric platform/dashboard allows users to securely organize, archive and share medical data such as x-rays, data from wearables, lab data, forms and appointments (analogous to Pinterest + Dropbox). Glimpse allows users to invite doctors, caretakers or loved ones to view their data securely. For providers, Lifespeed offers easy-to-use tools for digital content distribution, communication and analysis of patient engagement. The integration of consumer populated data with existing medical records creates an ecosystem that offers comprehensive collaboration between consumers and providers. The platform facilitates a marketplace where stakeholders and third party developers bring apps to the dashboard. The Lifespeed dashboard also provides a medium where healthcare organizations can advertise to targeted communities through the consumer centered portal for health data management. NICOlette NICOlette’s first offering is a forthcoming parent-facing, HIPAA-compliant app called NicoBoard. It shares NICU patients’ electronic health record (EHR) data in visually intuitive ways designed to allow parents to quickly grasp the information. It extracts the EHR raw data and synthesizes it into a consumable format for parents so that they can feel competent and confident in participating in their child’s journey to wellness. In addition to parsing out easily digestible EHR data visualizations (such as color-coded graphs of a patient’s oxygen saturation, carbon dioxide levels and apnea episode history, for example), NicoBoard will also furnish parents with curated research relevant to newborn health. The idea is for parents to use the research to make more informed healthcare decisions on behalf of their babies. SOFTWARE SOFTWARE Patient Engagement
  • 24. Thank You Leading Change for Better Pediatric Care