This corporate presentation provides an overview of Fibrocell Science, Inc., an autologous cell and gene therapy company. It summarizes Fibrocell's two lead product candidates, FCX-007 for Recessive Dystrophic Epidermolysis Bullosa and FCX-013 for Linear Scleroderma. For FCX-007, preclinical studies demonstrated expression of COL7A1 protein and anchoring fibrils in RDEB patient fibroblasts. A Phase I/II clinical trial is actively recruiting subjects with initial clinical data expected in 3Q17. For FCX-013, a proof-of-concept study in a mouse model showed reduction in dermal thickness. An IND filing is
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Cyclo Therapeutics: (OTCQB: CTDH) is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials. The Company’s Scientific Advisory Board endorsed Alzheimer’s Disease (AD) as the company’s next drug development indication. The Company was authorized by the FDA to use Trappsol® Cyclo™ for a late-onset AD patient under a Compassionate Use Program which began intravenous dosing in May 2018. Learn more at CTDHinfo.com.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing what it believes will be transformative therapies to overcome unmet medical needs in neurodegeneration and liver disease. The Company is developing NE3107 for Alzheimer’s (AD) and Parkinson’s (PD) and BIV201 for refractory ascites and HRS-AKI.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Cyclo Therapeutics: (OTCQB: CTDH) is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials. The Company’s Scientific Advisory Board endorsed Alzheimer’s Disease (AD) as the company’s next drug development indication. The Company was authorized by the FDA to use Trappsol® Cyclo™ for a late-onset AD patient under a Compassionate Use Program which began intravenous dosing in May 2018. Learn more at CTDHinfo.com.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing what it believes will be transformative therapies to overcome unmet medical needs in neurodegeneration and liver disease. The Company is developing NE3107 for Alzheimer’s (AD) and Parkinson’s (PD) and BIV201 for refractory ascites and HRS-AKI.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Posterior Segment Company Showcase - AGTCHealthegy
Posterior Segment Company Showcase - AGTC at OIS@AAO 2016.
Presenter:
Sue Washer, President & CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Tom Selleck Health: A Comprehensive Look at the Iconic Actor’s Wellness Journeygreendigital
Tom Selleck, an enduring figure in Hollywood. has captivated audiences for decades with his rugged charm, iconic moustache. and memorable roles in television and film. From his breakout role as Thomas Magnum in Magnum P.I. to his current portrayal of Frank Reagan in Blue Bloods. Selleck's career has spanned over 50 years. But beyond his professional achievements. fans have often been curious about Tom Selleck Health. especially as he has aged in the public eye.
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Introduction
Many have been interested in Tom Selleck health. not only because of his enduring presence on screen but also because of the challenges. and lifestyle choices he has faced and made over the years. This article delves into the various aspects of Tom Selleck health. exploring his fitness regimen, diet, mental health. and the challenges he has encountered as he ages. We'll look at how he maintains his well-being. the health issues he has faced, and his approach to ageing .
Early Life and Career
Childhood and Athletic Beginnings
Tom Selleck was born on January 29, 1945, in Detroit, Michigan, and grew up in Sherman Oaks, California. From an early age, he was involved in sports, particularly basketball. which played a significant role in his physical development. His athletic pursuits continued into college. where he attended the University of Southern California (USC) on a basketball scholarship. This early involvement in sports laid a strong foundation for his physical health and disciplined lifestyle.
Transition to Acting
Selleck's transition from an athlete to an actor came with its physical demands. His first significant role in "Magnum P.I." required him to perform various stunts and maintain a fit appearance. This role, which he played from 1980 to 1988. necessitated a rigorous fitness routine to meet the show's demands. setting the stage for his long-term commitment to health and wellness.
Fitness Regimen
Workout Routine
Tom Selleck health and fitness regimen has evolved. adapting to his changing roles and age. During his "Magnum, P.I." days. Selleck's workouts were intense and focused on building and maintaining muscle mass. His routine included weightlifting, cardiovascular exercises. and specific training for the stunts he performed on the show.
Selleck adjusted his fitness routine as he aged to suit his body's needs. Today, his workouts focus on maintaining flexibility, strength, and cardiovascular health. He incorporates low-impact exercises such as swimming, walking, and light weightlifting. This balanced approach helps him stay fit without putting undue strain on his joints and muscles.
Importance of Flexibility and Mobility
In recent years, Selleck has emphasized the importance of flexibility and mobility in his fitness regimen. Understanding the natural decline in muscle mass and joint flexibility with age. he includes stretching and yoga in his routine. These practices help prevent injuries, improve posture, and maintain mobilit
Flu Vaccine Alert in Bangalore Karnatakaaddon Scans
As flu season approaches, health officials in Bangalore, Karnataka, are urging residents to get their flu vaccinations. The seasonal flu, while common, can lead to severe health complications, particularly for vulnerable populations such as young children, the elderly, and those with underlying health conditions.
Dr. Vidisha Kumari, a leading epidemiologist in Bangalore, emphasizes the importance of getting vaccinated. "The flu vaccine is our best defense against the influenza virus. It not only protects individuals but also helps prevent the spread of the virus in our communities," he says.
This year, the flu season is expected to coincide with a potential increase in other respiratory illnesses. The Karnataka Health Department has launched an awareness campaign highlighting the significance of flu vaccinations. They have set up multiple vaccination centers across Bangalore, making it convenient for residents to receive their shots.
To encourage widespread vaccination, the government is also collaborating with local schools, workplaces, and community centers to facilitate vaccination drives. Special attention is being given to ensuring that the vaccine is accessible to all, including marginalized communities who may have limited access to healthcare.
Residents are reminded that the flu vaccine is safe and effective. Common side effects are mild and may include soreness at the injection site, mild fever, or muscle aches. These side effects are generally short-lived and far less severe than the flu itself.
Healthcare providers are also stressing the importance of continuing COVID-19 precautions. Wearing masks, practicing good hand hygiene, and maintaining social distancing are still crucial, especially in crowded places.
Protect yourself and your loved ones by getting vaccinated. Together, we can help keep Bangalore healthy and safe this flu season. For more information on vaccination centers and schedules, residents can visit the Karnataka Health Department’s official website or follow their social media pages.
Stay informed, stay safe, and get your flu shot today!
NVBDCP.pptx Nation vector borne disease control programSapna Thakur
NVBDCP was launched in 2003-2004 . Vector-Borne Disease: Disease that results from an infection transmitted to humans and other animals by blood-feeding arthropods, such as mosquitoes, ticks, and fleas. Examples of vector-borne diseases include Dengue fever, West Nile Virus, Lyme disease, and malaria.
Basavarajeeyam is a Sreshta Sangraha grantha (Compiled book ), written by Neelkanta kotturu Basavaraja Virachita. It contains 25 Prakaranas, First 24 Chapters related to Rogas& 25th to Rasadravyas.
Title: Sense of Taste
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the structure and function of taste buds.
Describe the relationship between the taste threshold and taste index of common substances.
Explain the chemical basis and signal transduction of taste perception for each type of primary taste sensation.
Recognize different abnormalities of taste perception and their causes.
Key Topics:
Significance of Taste Sensation:
Differentiation between pleasant and harmful food
Influence on behavior
Selection of food based on metabolic needs
Receptors of Taste:
Taste buds on the tongue
Influence of sense of smell, texture of food, and pain stimulation (e.g., by pepper)
Primary and Secondary Taste Sensations:
Primary taste sensations: Sweet, Sour, Salty, Bitter, Umami
Chemical basis and signal transduction mechanisms for each taste
Taste Threshold and Index:
Taste threshold values for Sweet (sucrose), Salty (NaCl), Sour (HCl), and Bitter (Quinine)
Taste index relationship: Inversely proportional to taste threshold
Taste Blindness:
Inability to taste certain substances, particularly thiourea compounds
Example: Phenylthiocarbamide
Structure and Function of Taste Buds:
Composition: Epithelial cells, Sustentacular/Supporting cells, Taste cells, Basal cells
Features: Taste pores, Taste hairs/microvilli, and Taste nerve fibers
Location of Taste Buds:
Found in papillae of the tongue (Fungiform, Circumvallate, Foliate)
Also present on the palate, tonsillar pillars, epiglottis, and proximal esophagus
Mechanism of Taste Stimulation:
Interaction of taste substances with receptors on microvilli
Signal transduction pathways for Umami, Sweet, Bitter, Sour, and Salty tastes
Taste Sensitivity and Adaptation:
Decrease in sensitivity with age
Rapid adaptation of taste sensation
Role of Saliva in Taste:
Dissolution of tastants to reach receptors
Washing away the stimulus
Taste Preferences and Aversions:
Mechanisms behind taste preference and aversion
Influence of receptors and neural pathways
Impact of Sensory Nerve Damage:
Degeneration of taste buds if the sensory nerve fiber is cut
Abnormalities of Taste Detection:
Conditions: Ageusia, Hypogeusia, Dysgeusia (parageusia)
Causes: Nerve damage, neurological disorders, infections, poor oral hygiene, adverse drug effects, deficiencies, aging, tobacco use, altered neurotransmitter levels
Neurotransmitters and Taste Threshold:
Effects of serotonin (5-HT) and norepinephrine (NE) on taste sensitivity
Supertasters:
25% of the population with heightened sensitivity to taste, especially bitterness
Increased number of fungiform papillae
Local Advanced Lung Cancer: Artificial Intelligence, Synergetics, Complex Sys...Oleg Kshivets
Overall life span (LS) was 1671.7±1721.6 days and cumulative 5YS reached 62.4%, 10 years – 50.4%, 20 years – 44.6%. 94 LCP lived more than 5 years without cancer (LS=2958.6±1723.6 days), 22 – more than 10 years (LS=5571±1841.8 days). 67 LCP died because of LC (LS=471.9±344 days). AT significantly improved 5YS (68% vs. 53.7%) (P=0.028 by log-rank test). Cox modeling displayed that 5YS of LCP significantly depended on: N0-N12, T3-4, blood cell circuit, cell ratio factors (ratio between cancer cells-CC and blood cells subpopulations), LC cell dynamics, recalcification time, heparin tolerance, prothrombin index, protein, AT, procedure type (P=0.000-0.031). Neural networks, genetic algorithm selection and bootstrap simulation revealed relationships between 5YS and N0-12 (rank=1), thrombocytes/CC (rank=2), segmented neutrophils/CC (3), eosinophils/CC (4), erythrocytes/CC (5), healthy cells/CC (6), lymphocytes/CC (7), stick neutrophils/CC (8), leucocytes/CC (9), monocytes/CC (10). Correct prediction of 5YS was 100% by neural networks computing (error=0.000; area under ROC curve=1.0).
micro teaching on communication m.sc nursing.pdfAnurag Sharma
Microteaching is a unique model of practice teaching. It is a viable instrument for the. desired change in the teaching behavior or the behavior potential which, in specified types of real. classroom situations, tends to facilitate the achievement of specified types of objectives.
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- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
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The Gram stain is a fundamental technique in microbiology used to classify bacteria based on their cell wall structure. It provides a quick and simple method to distinguish between Gram-positive and Gram-negative bacteria, which have different susceptibilities to antibiotics
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
2. This presentation and our accompanying remarks contain “forward-looking statements” within the meaning of the
U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified
as forward-looking statements for this purpose and include, among others, statements relating to: the potential
advantages of our product candidates; the initiation, design and timing of pre-clinical and clinical studies and
activities and the reporting of the results thereof; the timing of regulatory submissions and actions; expected
milestones; and all other statements relating to our future operations, future financial performance, future financial
condition, prospects or other future events.
Forward-looking statements are based upon our current expectations and assumptions and are subject to a number
of known and unknown risks, uncertainties and other factors that could cause actual results to differ materially and
adversely from those expressed or implied by such statements. Factors that could cause or contribute to such
differences include, among others: our ability to obtain additional capital to fund our operations; FDA allowance to
treat pediatric subjects in the Phase II portion of our Phase I/II clinical trial of FCX-007; uncertainties relating to the
initiation, enrollment and completion of clinical trials and whether the results will validate and support the safety
and efficacy of our product candidates; the risk that results seen in pre-clinical studies may not be replicated in
humans; varying interpretation of pre-clinical and clinical data; our ability to maintain our collaborations with
Intrexon; and the other factors discussed under the caption “Item 1A. Risk Factors” in our most recent Form 10-K
and Form 10-Q filings which are available through the “Investors—SEC Filings” page of our website at
www.fibrocell.com. As a result, you should not place undue reliance on forward-looking statements.
The forward-looking statements made in connection with this presentation represent our views only as of the date
of this presentation (or any earlier date indicated in such statement). While we may update certain forward-looking
statements from time to time, we specifically disclaim any obligation to do so, even if new information becomes
available in the future.
2
Forward-Looking Statements
3. Company Highlights
•Autologous cell and gene therapy company translating personalized
biologics into medical breakthroughs for diseases of the skin and
connective tissue
•FCX-007 — Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Actively recruiting adult subjects; first human clinical data expected in 3Q17
Granted pediatric rare disease designation by FDA
• FCX-013 — Linear Scleroderma
Proof-of-concept completed January 2016; IND planned 4Q17
Granted orphan drug designation by FDA for localized scleroderma
• Arthritis and related conditions
Deliver therapeutic protein locally to the joint providing sustained efficacy while
avoiding key side effects typically associated with systemic therapy
•Portfolio being developed in collaboration with Intrexon
3
4. Key Advantages of Our Autologous Fibroblast
Gene Therapy Platform
• Fibroblasts are the most common cell in skin and connective tissue, and are an ideal
delivery vehicle to administer protein of interest locally
Localized administration avoids side effects typically associated with systemic therapy
Reduced rejection and immunogenicity concerns because autologous fibroblasts are compatible
with the unique biology of each patient
• Use of a 3rd generation, self-inactivating (SIN) lentivirus:
Accommodates large gene constructs due to packaging size
Allows for target gene integration, ideal for long-term expression of the protein
• Fibroblasts are genetically modified ex vivo, providing distinct safety and efficacy
advantages:
Enables testing for safety and confirmation of transduction rates and protein expression prior to
administration
Reduces potential for host immune response upon retreatment as live virus is not administered
directly into the patient
• We are using our proprietary technology to create personalized biologics
Expertise in commercial-scale manufacturing of an autologous cell therapy
4
6. Development Pipeline
6
Personalized
Biologic Indication Research
Pre-Clinical
Development
Human Clinical
Trials
FCX-007
Recessive Dystrophic
Epidermolysis Bullosa (RDEB)
FCX-013 Linear Scleroderma
Research
Program
Arthritis
7. Recessive Dystrophic Epidermolysis Bullosa
7
Disease Current Treatments Epidemiology
• Cause: A mutation in the
COL7A1 gene that encodes
for COL7
• Devastating, progressive,
painful blistering disease
that often leads to death
• Diagnosed at infancy
• High mortality rate
• Current treatments only
address symptoms
Bandaging & antibiotics –
bandaging alone can
exceed $10,000 per
month1
Feeding tubes
Surgery, including hand
and esophageal
Dystrophic EB (DEB)
~5,500 – 12,500 US2
• RDEB
~1,100 – 2,500 US3
8. FCX-007 Providing Hope for RDEB Patients
8
RDEB patients do not produce
type VII collagen (COL7) due to
mutation in COL7A1 gene
Main component of anchoring
fibrils that connect skin layers
FCX-007 is an autologous human
dermal fibroblast transduced with
a lentiviral (LV) vector encoded for
COL7A1
Simple, local injection to the
papillary dermis
9. COL7 Expression Confirmation
9
Culture supernatant evaluated for in vitro COL7 expression
• ELISA assay indicates virus dose-dependent protein expression
• Trimeric form of COL7 produced by RDEB patient fibroblasts
transduced with LV-COL7
• Must be trimeric to be functional
Reference: Bruckner-Tuderman, Leena. Can Type VII Collagen Injections Cure Dystrophic
Epidermolysis Bullosa? Molecular Therapy (2008) 17 1, 6–7.
RDEB+Control
PurifiedCOL7
FCX-007-01
FCX-007-02
Trimeric
COL7
(900kDa)
COL7 IP
Immunoprecipitation (IP)/Western Blot
COL7 Formation
Trimeric Form
(900kDa)
10. 10
Detection of COL7 Expression by
Immunofluorescence (Magnification 20x)
• In vitro COL7 expression noted from FCX-007 replicates (red)
• FCX-007 expresses higher levels of COL7 than normal human
dermal fibroblasts (NHDF) as depicted by a brighter signal
• No expression of COL7 in RDEB-positive fibroblasts
FCX-007 Rep 1 FCX-007 Rep 2 FCX-007
In Vivo Expression*In Vitro Expression
NHDF RDEB+ NHDF RDEB+
• RDEB xenograft on immunodeficient mouse sampled 17 days
post-injection
• COL7 detected at the basement membrane zone (BMZ; refer to
arrow) and underlying dermis (green)
• NDHF slide expresses COL7 normally resulting in mature
anchoring fibrils
• No expression of COL7 in RDEB-positive fibroblasts*Image courtesy of Peter Marinkovich, MD lab, Stanford University
COL7 expression at
BMZ only 17 days
after injection of
FCX-007
11. • Autologous cells minimize rejection risk
• Direct administration of FCX-007 to the upper dermis ensures COL7
is expressed in the basement membrane zone where anchoring
fibrils are formed
• The integrative nature of the lentivirus allows the COL7A1 gene to
be passed to daughter cells as FCX-007 cells divide
• Academic research suggests that in vivo COL7 expression persists for
at least one year in RDEB skin tissue regenerated on
immunodeficient mice4
11
Long-lasting COL7 Persistence Expected
12. FCX-007 Phase I/II Clinical Trial Design
12
Title A Phase I/II Trial of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Objectives Primary
1) To evaluate the safety of FCX-007
Secondary
1) To evaluate mechanism of action of FCX-007 at weeks 12, 25, 52 and unscheduled visits through
the evaluation of skin biopsies for COL7 expression and the presence of anchoring fibrils
2) To evaluate the efficacy of FCX-007 through an intra-subject paired analysis of target wound area
at weeks 4, 12, 25, 52 and unscheduled visits, comparing FCX-007 treated wounds to untreated
wounds in Phase I and to wounds administered sterile saline in Phase II through the evaluation
of digital imaging of wounds
Number of
Subjects
Twelve subjects consisting of six adults in the Phase I portion of the trial and, subject to FDA
allowance, six pediatrics in the Phase II portion of the trial
Status Actively recruiting adult subjects in Phase I portion of trial
13. Linear Scleroderma
13
Disease Epidemiology
• Excess production of collagen
I and III characterized by skin
fibrosis and linear scars
• The linear areas of skin
thickening may extend to
underlying tissue and muscle
in children which may impair
growth in affected legs and
arms or forehead
• Lesions appearing across
joints impair motion and may
be permanent
• Localized Scleroderma
~160,000 sufferers US5
comprised of many different
sub-types
Linear Scleroderma
Initial target for FCX-013 is
a group of ~40,000
patients6
Current Treatments
Current treatments only
address symptoms:
• Systemic or topical
corticosteroids
• UVA light therapy
• Physical therapy
Photo: Reprinted from the Journal of the American Academy of Dermatology, Volume 59, Issue 3, Stéphanie Christen-Zaech, Miriam D. Hakim, F. Sule Afsar, Amy S. Paller.
Pediatric morphea (localized scleroderma): Review of 136 patients, Figure 1, pp. 385-396. Copyright Sept 2008. Used with permission from Elsevier Ltd.
14. FCX-013 Development Progressing
14
• Product profile
Autologous fibroblasts genetically modified using lentivirus and encoded for a
protein responsible for breaking down excess collagen I and III
Target protein known to breakdown collagens
Incorporates Intrexon’s RheoSwitch Therapeutic System® (RTS®) to control protein
expression
• RTS® has previous human clinical trial experience
• Proof-of-concept animal study data demonstrated protein expression,
reduced thickness of fibrotic tissue
• Scale-up manufacturing in process
• Received FDA orphan drug designation for treatment of localized
scleroderma
• Next steps include dose-ranging and toxicology/biodistribution studies
• IND submission expected 4Q17
15. FCX-013 Proof-of-Concept Study
15
• Study Design
Bleomycin treated SCID mouse model
N=30 mice over test and control groups
Assessed histologically for reduction of dermal
thickness and sub-dermal muscle in the presence
of FCX-013 and oral ligand
• Result
Bleomycin treatment resulted in skin fibrosis, measured by a significant increase in dermal thickness
Demonstrated that FCX-013 with ligand reduced the dermal thickness of fibrotic tissue to levels
similar to non-bleomycin (saline) with ligand treated skin
Further reduced the thickness of the sub-dermal muscle layer
Blecomycin treatments Ligand Treatment
D0 D28 D29 D39
Cell
injection
Harvest skin
samples
CONTROL:
Saline (no Bleo)
No Cells
TEST:
Bleomycin
FCX-013
CONTROL:
Bleomycin
Non-Modified Cells
16. • FCX-013 employs Intrexon’s RheoSwitch
Therapeutic System® (RTS®)
The RTS® biologic switch is activated by an orally-
administered activator ligand (AL) that provides the
ability to control level and timing of protein
expression
• Enhances the safety profile of FCX-013 by
providing control over the expression of the
protein
• In vitro studies support RTS® control over FCX-
013 protein expression:
Significant increase in expression of the target
protein in the presence of the AL
In the absence of the AL, the expression is reduced
to normal cellular production of the target protein
Cells transduced with RTS® and off-target gene
construct (RTS-GFP) and non-transduced fibroblasts
(mock) express normal cellular production levels
16
RheoSwitch® Control
RTS-GFP Control FCX-013 Mock Control
FCX-013 RTS® Control
no AL
+ AL
TargetProteinConcentration
17. Research Program for Arthritis
17
•Deliver therapeutic protein locally to the joint providing sustained
efficacy while avoiding key side effects typically associated with
systemic therapy
•Combines Fibrocell’s autologous fibroblast technology with
Intrexon’s cellular engineering to develop localized gene therapies
•Focused on addressing chronic inflammation and degenerative
diseases of the joint, including arthritis
19. Company Highlights
•Autologous cell and gene therapy company translating personalized
biologics into medical breakthroughs for diseases of the skin and
connective tissue
•FCX-007 — Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Actively recruiting adult subjects; first human clinical data expected in 3Q17
Granted pediatric rare disease designation by FDA
• FCX-013 — Linear Scleroderma
Proof-of-concept completed January 2016; IND planned 4Q17
Granted orphan drug designation by FDA for localized scleroderma
• Arthritis and related conditions
Deliver therapeutic protein locally to the joint providing sustained efficacy while
avoiding key side effects typically associated with systemic therapy
•Portfolio being developed in collaboration with Intrexon
19
20. References
1The Dystrophic Epidermolysis Research Association of America (DebRA). DEB
brochure, page 6: http://www.debra.org/pdfs/Debra-of-America-Brochure.pdf;
accessed 07/20/15.
2DEBRA International. What is EB Infographic: http://www.debra-
international.org/epidermolysis-bullosa.html.; accessed 10/06/2014.
3Petrof G., et al. Fibroblast cell therapy enhances initial healing in recessive
dystrophic epidermolysis bullosa wounds: results of a randomised, vehicle-
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