This document summarizes work by the MIT Center for Biomedical Innovation on analyzing the cell and gene therapy pipeline. It estimates that there will be approximately 80-100 new gene and cell therapy product approvals in the United States by 2031, treating around 410,000-550,000 patients. The majority of these therapies will be for oncology (cancer) patients. It also provides estimates of total reimbursement for these new therapies in 2031 of $20-30 billion, with $15-21 billion for oncology treatments. Near term therapies discussed include already approved CAR-T cancer therapies and gene therapies for rare diseases like spinal muscular atrophy.

1. MIT CENTER FOR BIOMEDICAL INNOVATION
Cell and Gene
Therapy Pipeline
19 November 2019
Danielle Rollmann
Senior Advisor, MIT FoCUS

2. MIT NEWDIGS – Helping the System Catch Up With the Science
• Safe haven “think & do” tank for
convening; MIT = trusted neutral
intermediary
• Track record of real world impact
• Interactive methods/tools for multi-
stakeholder collaboration
• Bold, transformational system
innovations for ten (10) years
2
NEWDIGS “Adaptive Licensing” Project fueled timely action &
impact in Europe from regulatory science innovation…..
… and Illuminated a Broad Set of Principles for Accelerating
Sustainable Patient-Centered Innovation

3. FoCUS: Multi-stakeholder group dedicated to making
innovative cures accessible and sustainable
MIT CENTER FOR BIOMEDICAL INNOVATION
>60 organizations,
170 individuals engaged
Patients Payers Hospitals
Developers Academics Policymakers
New
durable
therapies
Payer
coverage
Patient
access
• Growing clinical pipeline on
innovative durable therapies
• High one-time cost will likely
create financial challenges
• Sustainability for all
stakeholders is at risk
➢Need to address stakeholder
concerns, flesh out hurdles
and foster new tools

4. The “Pipeline Analysis” team
MIT CENTER FOR BIOMEDICAL INNOVATION
4
Colin Young, Ph.D
Jonathan Thomas, B.Sc
Jenniffer Bulovic, M.D.
Yu-Pu Wu, M.D.
Jonathan Salcedo, Ph.D candidate
Not shown here:
Dayton McMillan, M.D. candidate

5. Developing an estimate of the gene therapy pipeline
• ~1,300 active, US-based clinical
trials of durable cell and gene
therapies
• Expect ~80-100 product indications
by end 2031
• Product candidate mix
• ~58% for oncology patients
• ~27% for orphan, non-oncology
• ~15% for larger therapeutic areas
such as patients with cardiovascular
conditions
MIT CENTER FOR BIOMEDICAL INNOVATION

6. Indication-by-indication analysis
MIT CENTER FOR BIOMEDICAL INNOVATION
Schematic of Therapy Launch and Patient Number Estimation

7. Key uncertainty: Adoption rates
SMA
Haemophilia
R/R DLBCL
ALS
Diabetes 1
Macular
Degeneration
Parkinson’s
Retinitis
Pigmentosa
MIT CENTER FOR BIOMEDICAL INNOVATION
7
Fatal
Non fatal
Alternative
treatment
No alternative
treatment
In the model we make assumptions about
two parameters for each of incidence and
prevalence:
Peak penetration
What percentage of the clinically
eligible population will be treated
Time to peak
How long to achieve the peak
(from 1 to 7 years)
We are exploring the influence of disease
severity in determining model parameters
Key indicators
Slower Faster
Faster
Slower

8. US Model: Est. 80-100 new drug approvals by 2031
MIT CENTER FOR BIOMEDICAL INNOVATION
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410,000-550,000 patients treated by 2031
• 350,000-450,000 in oncology (40,000-50,000 in 2031)
• 60,000-100,000 in gene therapy (6,000-11,000 in 2031)
$20-30Bn total reimbursement in 2031
• $15-21Bn for oncological treatments
• $5-9Bn for gene therapies
$3,500B US Healthcare spending
(2017, National Health Expenditure Fact sheet)

9. Near term treatments (US)
Currently available
• Oncology CAR-T therapies: Kymriah®,
Yescarta®
• Currently approved for acute
lymphocytic leukemia (ALL) and
diffuse large B-cell lymphoma
(DLBCL)
• Ultra-rare disease treatments
• Luxturna®- for Retinitis pigmentosa &
Leber’s congenital amaurosis (both
RPE65)
• Zolgensma® - for Spinal muscular
atrophy
•
Other possible treatments within 5 years
• Hematological conditions
• Hemophilia A & B
• Sickle Cell anemia
• β-thalassemia
• Additional ophthalmological treatments
• RP & LCA (other genetic mutations)
• One or more other conditions possible
• Neurological conditions – one or more
• Higher prevalence disease treatments
• Macular degeneration possible
MIT CENTER FOR BIOMEDICAL INNOVATION

10. MIT CENTER FOR BIOMEDICAL INNOVATION
Cell and Gene
Therapy Pipeline
19 November 2019
Danielle Rollmann
Senior Advisor, MIT FoCUS
For more about FoCUS, please contact:
Karen Katz, J.D.
Director, FoCUS Program – Paying for Cures
MIT Center for Biomedical Innovation
New Drug Development Paradigms (NEWDIGS) Initiative
kkatz@mit.edu