cell, gene, & rna therapy landscape
TRANSCRIPT
Information Classification: General
Gene, Cell, & RNA Therapy Landscape
Q2 2021 Quarterly Data Report
Q2
20
21
Information Classification: General
About the authors
The American Society of Gene & Cell Therapy (ASGCT) is the primary professional membership organization for scientists, physicians, patient advocates, and other professionals with interest in gene and cell therapy.
Our members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. ASGCT advances knowledge, awareness, and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease to benefit patients and society.
Informa Pharma Intelligence powers a full suite of analysis products – DatamonitorHealthcare™, Sitetrove™, Trialtrove™, Pharmaprojects™, Biomedtracker™, Scrip™, Pink Sheet™ and In Vivo™ – to deliver the data needed by the pharmaceutical and biomedical industry to make decisions and create real-world opportunities for growth.
With more than 400 analysts, journalists, and consultants keeping their fingers on the pulse of the industry, no key disease, clinical trial, drug approval or R&D project isn’t covered through the breadth and depth of data available to customers. For more information visit pharmaintelligence.informa.com.
Table of contents04 Introduction
05 Key takeaways from Q2 2021
06 Key highlights in Q2 2021
11 Gene therapy pipeline
23 Non-genetically modified cell therapy pipeline
29 RNA therapy pipeline
35 Overview of dealmaking
38 Start-up funding
43 Upcoming catalysts
45 Appendix
Information Classification: General
4 / Q2 2021
Introduction
ASGCT is pleased to provide the second in a quarterly report series with our data partner, Informa Pharma Intelligence. This series provides an iterative, detailed look at progress in the preclinical and clinical development landscape of gene and cell therapies globally, and should collectively tell a complete story of scientific achievement and clinical advancement.
Highlights in Q2 2021 include the approval of a new gene therapy in Japan, the expansion of gene therapy approvals to three new countries, continued growth in early-phase gene therapy development, and growth in studies using a herpes simplex virus.
The dollar value of start-up financing rebounded this quarter to an aggregate of $1.4 billion after a consecutive three-quarter decrease.
You will find new types of information in this quarter’s report, as well, such as new clinical trials, key regulatory events, and listings of therapies in the preregistration phase this quarter, plus changes from Q2 2020 and noteworthy upcoming approval decisions.
ASGCT is excited to continue this series of landscape reports and to tell the story of this emerging technology as it translates to clinics and improves the lives of patients worldwide.
Information Classification: General
5 / Q2 2021
Key takeaways from Q2 2021
One new gene therapy was approved, and three previously-approved gene therapies were approved in additional countries/regions
• Delytact, a new genetically engineered oncolytic herpes simplex virus type 1, was approved in Japan for malignant glioma
• Yescarta gained approval in China, Zolgensma in South Korea, and Abecma in Canada
Oncology remains the most active therapeutic area of research (preclinical through preregistration)
• More than 1,300 gene and cell therapy candidates are in development for oncology
Financing for start-ups showed a marked increase
• Seed and Series A financing volume nearly doubled in Q2, and the dollar value tripled, due to four $100M+ fundraisers
Overall dealmaking volume was down from Q1 but up from a year ago
• A total of 142 deals (financings, alliances, and acquisitions) were signed in Q2, down from 167 in Q1, but up from the 132 transactions in Q2 2020
Information Classification: General
7 / Q2 2021
Approved gene therapies as of Q2 2021
Source: Pharmaprojects | Informa, July 2021
Product name Generic nameYear first approved
Disease(s) Locations approved Originator company
Gendicine recombinant p53 gene 2004 Head and neck cancer China Shenzhen SiBiono GeneTech
Oncorine E1B/E3 deficient adenovirus 2005Head and neck cancer; nasopharyngeal cancer
China Shanghai Sunway Biotech
Rexin-G mutant cyclin-G1 gene 2006 Solid tumors Philippines Epeius Biotechnologies
Neovasculgenvascular endothelial growth factor gene
2011Peripheral vascular disease; limb ischemia
Russian Federation, Ukraine Human Stem Cells Institute
Imlygic talimogene laherparepvec 2015 Melanoma US, EU, Australia Amgen
Strimvelis autologous CD34+ enriched cells 2016 Adenosine deaminase deficiency EU Orchard Therapeutics
Kymriah tisagenlecleucel-t 2017Acute lymphocytic leukemia; diffuse large B-cell lymphoma
US, EU, Japan, Australia, Canada, South Korea
Novartis
Luxturna voretigene neparvovec 2017Leber’s congenital amaurosis; retinitis pigmentosa
US, EU, Australia, Canada Spark Therapeutics (Roche)
Yescarta axicabtagene ciloleucel 2017Diffuse large B-cell lymphoma; non-Hodgkin’s lymphoma; follicular lymphoma
US, EU, Japan, Canada, China Kite Pharma (Gilead)
Collategene beperminogene perplasmid 2019 Critical limb ischemia Japan AnGes
Zolgensma onasemnogene abeparvovec 2019 Spinal muscular atrophyUS, EU, Japan, Australia, Canada, Brazil, Israel, Taiwan, South Korea
Novartis
Zynteglo lentiviral beta-globin gene transfer 2019Transfusion-dependent beta thalassemia
EU bluebird bio
Tecartus brexucabtagene autoleucel 2020 Mantel cell lymphoma US, EU Kite Pharma (Gilead)
Libmeldy OTL-200 2020 Metachromatic Leukodystrophy EU Orchard Therapeutics
Breyanzi lisocabtagene maraleucel 2021 Diffuse large B-cell lymphoma US, Japan Celgene (Bristol Myers Squibb)
Abecma idecabtagene vicleucel 2021 Multiple myeloma US, Canada bluebird bio
Delytact teserpaturev 2021 Malignant Glioma Japan Daiichi Sankyo
Text highlighted in yellow represent new approvals during Q2 2021
Information Classification: General
8 / Q2 2021
Approved RNA therapies as of Q2 2021 (1/2)
Source: Pharmaprojects | Informa, July 2021 Text highlighted in yellow represent new approvals during Q2 2021
Product name Generic nameYear first approved
Disease(s) Locations approved* Originator company
Kynamro mipomersen sodium 2013 Homozygous familial hypercholesterolaemiaUS, Mexcio, Argentina, South Korea
Ionis Pharmaceuticals
Exondys 51 eteplirsen 2016 Dystrophy, Duchenne muscular US Sarepta Therapeutics
Spinraza nusinersen 2016 Muscular atrophy, spinal
US, EU, Canada, Japan, Brazil, Switzerland, Australia, South Korea, China, Argentina, Colombia, Taiwan,
Ionis Pharmaceuticals
Tegsedi inotersen 2018 Amyloidosis, transthyretin-related hereditary EU, Canada, US, Brazil Ionis Pharmaceuticals
Onpattro patisiran 2018 Amyloidosis, transthyretin-related hereditaryUS, EU, Japan, Canada, Switzerland, Brazil, Taiwan
Alnylam
Vyondys 53 golodirsen 2019 Dystrophy, Duchenne muscular US Sarepta Therapeutics
Waylivra volanesorsen 2019 Hypertriglyceridaemia; Lipoprotein lipase deficiency EU, UK Ionis Pharmaceuticals
Comirnaty tozinameran 2020 Infection, coronavirus, novel coronavirus prophylaxis
UK, Bahrain, Israel, Canada, US, Mexico, Kuwait, Singapore, Saudi Arabia, Chile, Switzerland, EU, Colombia, Philippines, Australia, Hong Kong, Peru, South Korea, New Zealand, Japan, Brazil, Sri Lanka, Vietnam, Thailand, Oman,
BioNTech
*For COVID-19 vaccines, this includes emergency use authorization and full approvals
Information Classification: General
9 / Q2 2021
Approved RNA therapies as of Q2 2021 (2/2)
Source: Pharmaprojects | Informa, July 2021 Text highlighted in yellow represent new approvals during Q2 2021
Product name Generic nameYear first approved
Disease(s) Locations approved* Originator company
Moderna COVID-19 vaccine
COVID-19 vaccine, Moderna 2020 Infection, coronavirus, novel coronavirus prophylaxis
US, Canada, Israel, EU, Switzerland, Singapore, Qatar, Vietnam, UK, Philippines, Thailand, Japan, South Korea, Brunei, Paraguay, Taiwan, Botswana, India, Indonesia, Saudi Arabia
Moderna Therapeutics
Givlaari givosiran 2020 Porphyria US, EU, Canada, Switzerland Alnylam
Oxlumo lumasiran 2020 Hyperoxaluria EU, US Alnylam
Ampligen rintatolimod 2020 Chronic fatigue syndrome Argentina AIM ImmunoTech
Viltepso viltolarsen 2020 Dystrophy, Duchenne muscular US, Japan NS Pharma
Leqvio inclisiran 2020Atherosclerosis; Heterozygous familial hypercholesterolaemia; Hypercholesterolaemia; Homozygous familial hypercholesterolaemia
EU, UK Alnylam
Amondys 45 casimersen 2021 Dystrophy, Duchenne muscular US Sarepta Therapeutics
*For COVID-19 vaccines, this includes emergency use authorization and full approvals
Information Classification: General
10 / Q2 2021
Key highlights in Q2 2021
Below are select noteworthy events that happened in Q2 2021
Source: Biomedtracker | Informa, July 2021
Brand Name Generic Drug Name Molecule type Disease Event Type Event Date
Zynteglo betibeglogene autotemcel Viral Gene Therapy Transfusion-dependent β-thalassemia Withdrawal from German Market Apr 20, 2021
Delytact teserpaturev Viral Malignant glioma Approval (Japan) Jun 11, 2021
Roctavian valoctocogene roxaparvovecViral Gene Therapy
Hemophilia A MAA Submission (Europe) Jun 28, 2021
N/A ciltacabtagene autoleucel Cellular* Multiple myeloma MAA Submission (Europe) Apr 30, 2021
Abecma idecabtagene vicleucel Cellular* Multiple myeloma CHMP (European Panel) Results (Positive) Jun 24, 2021
Skysona elivaldogene autotemcel Viral Gene Therapy Cerebral adrenoleukodystrophy CHMP (European Panel) Results (Positive) May 20, 2021
N/A ALLO-715 Cellular* Multiple myelomaRegenerative Medicine Advanced Therapy (RMAT) Designation
Apr 21, 2021
N/A AK-OTOF Viral Gene Therapy Hearing loss Rare Pediatric Disease (RPD) Designation Apr 13, 2021
N/A BBP-631 Viral Gene Therapy Congenital Adrenal Hyperplasia (CAH) Rare Pediatric Disease (RPD) Designation May 14, 2021
N/A LX-2006 Viral Gene Therapy Friedreich's Ataxia Rare Pediatric Disease (RPD) Designation Jun 30, 2021
*These are genetically modified cell therapies (CAR-T therapies)
Information Classification: General
Q2
20
21Gene therapy pipeline
Gene therapy and genetically modified cell therapies
Information Classification: General
12 / Q2 2021
Gene therapy pipeline
• In May 2021, there were 1,745 therapies in development from preclinical through preregistration.
• Preclinical therapies account for the majority of the pipeline with 1,223 therapies in preclinical studies. This accounts for 70% of therapies in clinical development.
Source: Pharmaprojects | Informa, May 2021
0
200
400
600
800
1000
1200
1400
1600
1800
2000
Number of Therapies by Year
Gene Therapy Pipeline updated to include 2021 data*
Preclinical Phase I Clinical Trial Phase II Clinical Trial Phase III Clinical Trial Pre-registration
*These snapshots are taken every May to allow for year-on-year comparisons
Information Classification: General
13 / Q2 2021
Global Status as of May
Number of Therapies
2020 2021
Preclinical 911 1,223
Phase I Clinical Trial 180 254
Phase II Clinical Trial 199 234
Phase III Clinical Trial 24 26
Pre-registration 7 8
Total number of therapies (preclinical to pre-registration) 1,321 1,745
Total number of therapies in the clinic 410 522
Gene therapy pipeline – 2020 vs 2021
• When comparing preclinical development between 2020 and 2021, there was a significant increase in the number of therapies in preclinical development with 911 in 2020 and 1,223 therapies in 2021
• There was a significant increase in Phase I clinical trials in 2021 (from 180 therapies in 2020 vs 254 therapies in 2021)
Source: Pharmaprojects | Informa, May 2021
0
200
400
600
800
1000
1200
1400
Preclinical Phase I Clinical Trials Phase II Clinical Trials Phase III Clinical Trials
Number of therapies in development as of May
2020 2021
Information Classification: General
14 / Q2 2021
Gene therapy pipeline – Q1 vs Q2 2021
• Over the course of the second quarter, the largest growth was a 9 percent increase in the number of therapies in preclinical development
• The number of Phase III therapies remained the same, and there is one less therapy in pre-registration with the approval of Delytact
• Therapies currently in preregistration:
• valoctocogene roxaparvovec (Biomarin)
• lenadogene nolparvovec (Genethon, GenSight Biologics)
• elivaldogene autotemcel (Bluebird Bio)
• nadofaragene firadenovec (Ferring, FKD Therapeutics, Trizell)
• relmacabtagene autoleucel (JW Therapeutics)
• ciltacabtagene autoleucel (Johnson & Johnson)
• eladocagene exuparvovec (PTC Therapeutics)
Source: Pharmaprojects | Informa, May 2021
Global Status April 2021 July 2021
Preclinical 1,190 1,296
Phase I Clinical Trials 225 269
Phase II Clinical Trials 231 236
Phase III Clinical Trials 27 27
Pre-registration 8 7
Total 1,711 1,835
Information Classification: General
15 / Q2 2021
Gene therapy pipeline: Most commonly targeted therapeutic areas
• The top 2 therapeutic areas targeted by gene therapies from preclinical through pre-registration are oncology and rare diseases. Rare diseases includes oncology rare diseases
• The focus on oncology and rare diseases remains for therapies in clinical development
Source: Pharmaprojects | Informa, July 2021
1048
869
202
139
135
76
71
53
52
49
39
29
26
7
4
1
Anticancer Products
Rare Diseases
Neurological Products
Alimentary/Metabolic Products
Sensory Products
Blood and Clotting Products
Musculoskeletal Products
Anti-infective Products
Immunological Products
NA/Unspecified
Cardiovascular Products
Respiratory Products
Dermatological Products
Genitourinary (including sex hormones)
Miscellaneous Products
Hormonal Products (excluding sex hormones)
Number of therapies
Number of therapies from preclinical through pre-registration
375
335
35
31
30
24
17
16
15
13
10
3
2
2
1
Anticancer Products
Rare Diseases
Alimentary/Metabolic Products
Sensory Products
Blood and Clotting Products
Neurological Products
Musculoskeletal Products
Immunological Products
Anti-infective Products
Cardiovascular Products
Dermatological Products
Genitourinary (including sex hormones)
Miscellaneous Products
Respiratory Products
NA/Unspecified
Number of therapies
Therapies in the clinic (excludes preclinical therapies)
Information Classification: General
16 / Q2 2021
275
157
118
107
83
82
70
69
57
55
54
52
44
43
42
38
36
35
35
33
Cancer, solid, unspecified
Cancer, unspecified
Cancer, myeloma
Cancer, leukaemia, acute lymphocytic
Cancer, lymphoma, non-Hodgkin's
Cancer, leukaemia, acute myelogenous
Cancer, haematological, unspecified
Cancer, lymphoma, B-cell
Cancer, pancreatic
Cancer, liver
Cancer, ovarian
Cancer, breast
Cancer, lung, non-small cell
Cancer, colorectal
Cancer, brain
Cancer, leukaemia, chronic lymphocytic
Cancer, melanoma
Cancer, gastrointestinal, stomach
Cancer, lung, unspecified
Cancer, leukaemia, unspecified
Number of therapies
Gene therapy pipeline: Most common oncology diseases targeted• From preclinical through pre-registration, 1,048 therapies are being developed for oncology• Multiple myeloma and acute lymphocytic leukemia (ALL) remain the top two specified oncology indications• Non-Hodgkin’s lymphoma is now the third most common indication (acute myelogenous leukemia in Q1)
Source: Pharmaprojects | Informa, July 2021
Information Classification: General
17 / Q2 2021
27
21
19
19
18
16
16
15
12
11
11
11
10
9
8
8
7
7
6
6
Retinitis pigmentosa
Haemophilia A
Amyotrophic lateral sclerosis
Dystrophy, Duchenne's…
Anaemia, sickle cell
Cystic fibrosis
Haemophilia B
Thalassaemia
Batten's disease
Gaucher's disease
Leber's congenital amaurosis
Usher syndrome
Fabry's disease
Huntington's disease
Dementia, frontotemporal
Pompe's disease
Hyperphenylalaninaemia
Rett Syndrome
Angelman syndrome
Charcot–Marie–Tooth disease
Number of therapies
Non-oncology rare diseases
Gene therapy pipeline: Most common non-oncology rare diseases targeted
• There are 869 therapies in development for rare diseases from preclinical through pre-registration. This number includes oncology rare diseases. There are 435 therapies for non-oncology rare diseases in development
• Retinitis pigmentosa, Hemophilia A, amyotrophic lateral sclerosis (ALS), Duchenne’s muscular dystrophy (DMD), and sickle cell anemia make up the top 5 non-oncology rare diseases most targeted by gene therapies
Source: Pharmaprojects | Informa, July 2021
Information Classification: General
18 / Q2 2021
Gene therapy pipeline: Most common additional diseases targeted
• From preclinical through pre-registration, there are 356 therapies that are in development for diseases outside of oncology or rare diseases
• Of diseases that are not oncology or rare diseases that are disclosed: neurological diseases, ocular disorders, Alzheimer’s disease, HIV/AIDs, and Parkinson’s Disease make up the 5 diseases most targeted by gene therapies
Source: Pharmaprojects | Informa, July 2021
48
42
25
13
13
13
12
9
9
8
8
7
7
7
7
6
6
6
6
6
Undisclosed
Neurological disease, unspecified
Ocular disorder, unspecified
Alzheimer's disease
Infection, HIV/AIDS
Parkinson's disease
Hearing loss
Diabetes, Type 1
Macular degeneration, age-related, wet
Autoimmune disease, unspecified
Macular degeneration, age-related, dry
Arthritis, osteo
Cardiovascular disease, unspecified
Dermatological disease, unspecified
Musculoskeletal disease, unspecified
Hepatic dysfunction, alpha-1 antitrypsin…
Hepatic dysfunction, unspecified
Infection, coronavirus, novel coronavirus
Infection, hepatitis-B virus
Macular dystrophy, Stargardt's
Number of therapies
Non-oncology & non-rare diseases targeted by gene therapies
Information Classification: General
19 / Q2 2021
Gene therapy pipeline: Most common targets
Of the gene therapies in preclinical through pre-registration in which targets are disclosed:
• CD19 and B-cell maturation antigen (BCMA), also known as TNF receptor superfamily member 17, remain the most common targets for oncology indications
• Coagulation factor VIII is the most common target for non-oncology indications
Source: Pharmaprojects | Informa, July 2021
14
9
8
8
7
6
6
6
5
5
5
5
5
5
4
4
4
4
coagulation factor VIII
glucosylceramidase beta
coagulation factor IX
vascular endothelial growth factor A
CF transmembrane conductance regulator
dystrophin
hemoglobin subunit beta
N-acetyl-alpha-glucosaminidase
ATP binding cassette subfamily A member 4
collagen type VII alpha 1 chain
galactosidase alpha
hemoglobin subunit gamma 1
N-sulfoglucosamine sulfohydrolase
serpin family A member 1
C-C motif chemokine receptor 5 (gene/pseudogene)
gap junction protein beta 2
hemoglobin subunit gamma 2
iduronidase alpha-L-
Number of therapies
Non-oncology targets
147
64
37
27
22
21
21
17
14
14
14
12
11
11
10
10
10
10
9
CD19 molecule
TNF receptor superfamily member 17
CD22 molecule
Not applicable
erb-b2 receptor tyrosine kinase 2
membrane spanning 4-domains A1
mesothelin
glypican 3
cancer/testis antigen 1B
interleukin 3 receptor subunit alpha
programmed cell death 1
CD33 molecule
CD7 molecule
killer cell lectin like receptor K1
claudin 18
epidermal growth factor receptor
mucin 1, cell surface associated
Not available on LocusLink/Entrez Gene
CD38 molecule
Number of therapies
Oncology targets
Information Classification: General
20 / Q2 2021
Gene therapy clinical trial activity in 2021
• A total of 75 clinical trials were initiated in 2021 so far
• The top 10 diseases for trial starts in 2021 were all for oncology with the top 3 being the blood cancers non-Hodgkin’s lymphoma (NHL), acute lymphocytic leukemia (ALL), and multiple myeloma
Source: Trialtrove | Informa, July 2021
0
5
10
15
20
25
30
35
I I/II II III Other
Number of trials by phase of study22
12
10
6
4
4
4
4
4
3
3
3
2
2
2
2
2
2
2
2
Oncology: Lymphoma, Non-Hodgkin's
Oncology: Leukemia, Acute Lymphocytic
Oncology: Multiple Myeloma
Oncology: Unspecified Solid Tumor
Oncology: Breast
Oncology: Cervical
Oncology: Head/Neck
Oncology: Leukemia, Acute Myelogenous
Oncology: Lymphoma, Hodgkin's
Oncology: Bladder
Oncology: Liver
Oncology: Lung, Non-Small Cell
Autoimmune/Inflammation: Epidermolysis Bullosa
Infectious Disease: HPV
Metabolic/Endocrinology: Sickle Cell Disease
Oncology: Colorectal
Oncology: Esophageal
Oncology: Gastric
Oncology: Leukemia, Chronic Lymphocytic
Oncology: Ovarian
Number of trials
Trials by Disease
Information Classification: General
21 / Q2 2021
Gene therapy pipeline: Most commonly used vectors
• 89% of gene therapies in development use viral vectors for delivery• Adeno-associated virus (AAV) and lentivirus remain most common viral vectors used in development • The largest quarterly growth: 33 percent increase in studies using a herpes simplex virus
Source: Cell and Gene Therapy dashboard | Informa, July 2021
405
288
123
73
3624 15
0
50
100
150
200
250
300
350
400
450
Adeno-associatedvirus (AAV)
Lentivirus Adenovirus Retrovirus Herpes simplex virus Poxivirus Other viruses
Viral vectors used in gene therapies
Information Classification: General
22 / Q2 2021
Gene therapy breakdown: CAR Ts dominate pipeline
▪ Of the gene therapies in development, 68% are genetically modified cell therapies
▪ Chimeric Antigen Receptor T cells (CAR Ts) represent 49% while T cell receptor T cells (TCR Ts) account for 12% of the genetically modified cell therapies in development
22
Gene therapy pipeline
Genetically modified cell therapies Gene therapy
Genetically modified cell therapy breakdown
CAR-T CAR-NK TCR-T Other
Source: Cell and Gene Therapy dashboard | Informa, July 2021
Information Classification: General
24 / Q2 2021
Non-genetically modified cell therapy pipeline
• In May 2021, there were 771 therapies in development (preclinical through pre-registration):
• 64% of candidates are in preclinical development
• There were 33 therapies in Phase III clinical studies
• There are 4 therapies in pre-registration (undergoing regulatory review):
• Gliovac (Epitopoietic Research)
• Astrostem (K-StemCell/Nature Cell)
• Donislecel (CellTrans)
• RVT-802 (Enzyvant Sciences/SinovantSciences)
Source: Pharmaprojects | Informa, May 2021
0
100
200
300
400
500
600
700
800
900
Number of Therapies by Year
Non-genetically modified cell therapy pipeline*
Preclinical Phase I Clinical Trial Phase II Clinical Trial
Phase III Clinical Trial Pre-registration
*These are snapshots taken every May to allow for year-on-year comparisons
Information Classification: General
25 / Q2 2021
278
273
117
109
108
88
75
58
50
48
45
40
31
29
26
4
Anticancer Products
Rare Diseases
Neurological Products
Musculoskeletal Products
Alimentary/Metabolic Products
Cardiovascular Products
Immunological Products
Respiratory Products
Dermatological Products
Miscellaneous Products
Sensory Products
Anti-infective Products
Genitourinary (including sex hormones)
Blood and Clotting Products
NA/Unspecified
Hormonal Products (excluding sex hormones)
Number of therapies
Non-genetically modified cell therapy pipeline: Most common therapeutic areas targetedOf the cell therapies in development (preclinical through pre-registration):
• Similar to gene therapy, oncology and rare diseases are the most targeted therapeutic areas for non-genetically modified cell therapies (rare diseases includes oncology rare diseases).
Source: Pharmaprojects | Informa, July 2021
Information Classification: General
26 / Q2 2021
Non-genetically modified cell therapy pipeline: Most common cancers targeted
Of the cell therapies in development (preclinical through pre-registration) for oncology:
• Of the diseases in which indications are specified, the top three indications are brain cancer, ovarian cancer and non-small cell lung cancer
Source: Pharmaprojects | Informa, July 2021
9653
2925
2423
212121
1818
1713
1210
977
66
Cancer, solid, unspecifiedCancer, unspecified
Cancer, brainCancer, ovarian
Cancer, lung, non-small cellCancer, leukaemia, acute myelogenous
Cancer, haematological, unspecifiedCancer, liver
Cancer, melanomaCancer, head and neck
Cancer, pancreaticCancer, myeloma
Myelodysplastic syndromeCancer, breast
Cancer, colorectalCancer, prostate
Cancer, lymphoma, unspecifiedCancer, renal
Cancer, cervicalCancer, gastrointestinal, stomach
Number of therapies
Information Classification: General
27 / Q2 2021
Non-genetically modified cell therapy pipeline: Most common rare diseases targeted
Of the cell therapies in development (preclinical through pre-registration) for non-oncology rare diseases:
• Rare diseases: second most targeted therapeutic category for non-genetically modified cell therapies
• Of the non-oncology rare diseases being targeted, the top 3 are respiratory distress syndrome (ARDS), graft-versus-host disease (GVHD), and spinal cord injury
Source: Pharmaprojects | Informa, July 2021
3825
2418
12888
66
5444
333333
Respiratory distress syndrome, acuteGraft-versus-host disease
Spinal cord injuryAmyotrophic lateral sclerosis
Cytokine release syndromeDystrophy, Duchenne's muscular
Huntington's diseaseRetinitis pigmentosaAnaemia, sickle cell
SclerodermaFibrosis, pulmonary, idiopathic
Buerger's syndromeEpidermolysis bullosa
Neuritis, opticAcute lung injury
Anaemia, aplastic, unspecifiedAnal fistula
Dementia, frontotemporalHaemophilia A
Hypoplastic left heart syndrome
Number of therapies
Information Classification: General
28 / Q2 2021
Non-genetically modified cell therapy trial activity in 2021
Source: Trialtrove | Informa, July 2021
• A total of 40 clinical trials were initiated in 2021 so far
• The top four diseases for trial starts in 2021 were for COVID-19 infections, osteoarthritis, ovarian cancer, fallopian tube cancer and Parkinson’s disease
0
2
4
6
8
10
12
14
I II I/II III II/III Other
Number of trials by phase of study8
4
4
3
2
2
2
2
2
1
1
1
1
1
1
1
1
1
1
1
Novel coronavirus (2019-nCoV, COVID-19)
Osteoarthritis
Oncology: Ovarian
Oncology: Fallopian Tube
Parkinson's Disease
Cytomegalovirus Infection (CMV)
Respiratory Infections
Oncology: Multiple Myeloma
Oncology: Primary Peritoneal
Atopic Dermatitis
Cytokine Release Syndrome (CRS)
Epidermolysis Bullosa
Rheumatoid Arthritis
Transplantation/GVHD
Congestive Heart Failure
Alzheimer's Disease
Amyotrophic Lateral Sclerosis
Autism
Cerebral Palsy
Movement Disorders Number of trials
Trials by Disease
Information Classification: General
30 / Q2 2021
RNA therapies pipeline
• In 2021:
• More than 600 therapies were in development (from preclinical to pre-registration stage), with 73% of therapies in preclinical development
• 20 therapies were in Phase III clinical studies
• 1 therapy in pre-registration (vutrisiran, Alnylam)
Source: Pharmaprojects | Informa, May 2021
0
100
200
300
400
500
600
700
Nu
mb
er o
f th
erap
ies
RNA therapies pipeline*
Preclinical Phase I Clinical Trial Phase II Clinical Trial Phase III Clinical Trial Pre-registration
*These are snapshots are taken every May to allow for year-on-year comparisons
Information Classification: General
31 / Q2 2021
RNA therapy pipeline: Most common modalities
203
188
183
86
70
63
58
RNA interference
Antisense therapy
Messenger RNA
Anticancer, other
Prophylactic vaccine, anti-infective
Anticancer, immunological
Oligonucleotide, non-antisense, non-…
Number of therapies
April 2021
Source: Pharmaprojects | Informa, July 2021
• Between Q1 and Q2 2021, messenger RNA therapeutics overtook RNA interference (RNAi) and antisense oligonucleotides to become the most common modality in development (preclinical through pre-registration
219
211
191
84
79
78
59
Messenger RNA
RNA interference
Antisense therapy
Anticancer, other
Prophylactic vaccine, anti-infective
Anticancer, immunological
Oligonucleotide, non-antisense,…
Number of therapies
July 2021
Information Classification: General
32 / Q2 2021
RNA therapies: Most common diseases targeted
Of the 692 RNA therapies currently in the pipeline (from preclinical through pre-registration):
• Oncology and rare diseases are the top 2 therapeutic areas being targeted. However, unlike gene therapies and non-genetically modified cell therapies, the top therapeutic area being targeted by RNA therapeutics is for rare diseases (please note that rare oncology diseases are included in the rare diseases therapeutic category)
241
173
142
96
94
59
47
44
42
37
25
19
17
16
7
3
2
Rare Diseases
Anticancer Products
Anti-infective Products
Neurological Products
Alimentary/Metabolic Products
Musculoskeletal Products
Sensory Products
Respiratory Products
Cardiovascular Products
NA/Unspecified
Immunological Products
Blood and Clotting Products
Genitourinary (including sex hormones)
Dermatological Products
Miscellaneous Products
Antiparasitic Products
Hormonal Products (excluding sex hormones)
Number of therapiesSource: Pharmaprojects | Informa, July 2021
Information Classification: General
33 / Q2 2021
RNA therapies: Most common diseases targetedOf the RNA therapies currently in the pipeline (from preclinical through pre-registration):
• Top oncology indications targeted are solid tumors with the top 5 (where specified) being melanoma, breast cancer, liver cancer, non-small cell lung cancer (NSCLC), and pancreatic cancer
• For non-oncology rare diseases, Duchenne’s muscular dystrophy, cystic fibrosis, Huntington’s disease, amyotrophic lateral sclerosis (ALS), and retinitis pigmentosa are the top 5 targeted diseases
Source: Pharmaprojects | Informa, July 2021
5452
2520202020
181515
1313
76
55555
4
Cancer, solid, unspecifiedCancer, unspecifiedCancer, melanoma
Cancer, breastCancer, liver
Cancer, lung, non-small cellCancer, pancreaticCancer, colorectal
Cancer, head and neckCancer, prostate
Cancer, brainCancer, ovarian
Cancer, renalCancer, bladder
Cancer, leukaemia, acute myelogenousCancer, lymphoma, unspecified
Cancer, mesotheliomaCancer, myeloma
Cancer, skin, unspecifiedCancer, lung, unspecified
Number of therapies
Oncology indications23
109
88
66
5555
444
333333
Dystrophy, Duchenne's muscularCystic fibrosis
Huntington's diseaseAmyotrophic lateral sclerosis
Retinitis pigmentosaDystrophy, myotonic muscular
Respiratory distress syndrome, acuteAngelman syndrome
COVID-19 complicationsFibrosis, pulmonary, idiopathic
Primary ciliary dyskinesiaAmyloidosis, transthyretin-related hereditary
HypertriglyceridaemiaLipoprotein lipase deficiency
Anaemia, sickle cellAngioedema, hereditary
Ataxia, spinocerebellarChronic obstructive pulmonary disease
Dystrophy, facioscapulohumeral muscularHyperphenylalaninaemia
Number of therapies
Non-oncology rare diseases
Information Classification: General
34 / Q2 2021
RNA therapy pipeline: Clinical trial activity
• 162 RNA therapy trials were initiated in the first 6 months of 2021
• Trials evaluating vaccines for COVID-19 infections predominate but there are also trials initiated for dyslipidemia and hepatitis B infections
Source: Trialtrove | Informa, July 2021
0
10
20
30
40
50
60
II IV I III I/II II/III Other
Number of trials initiated by phase 102
9
7
4
3
3
3
3
2
2
2
2
2
1
1
1
1
1
1
Novel coronavirus (2019-nCoV, COVID-19)
Dyslipidemia
Infectious Disease: HBV
Oncology: Unspecified Solid Tumor
Hepatic Fibrosis
Hypertension
Amyotrophic Lateral Sclerosis
NAFLD
Muscular Dystrophy
Spinal Muscular Atrophies
Respiratory Infections
Oncology: Prostate
Vaccines (Infectious Disease): Other Viral Vaccines
Sjogren's Syndrome
Acute Coronary Syndromes
Congestive Heart Failure
Coronary Artery Disease
Peripheral Arterial Disease
Thrombotic Disorders
Number of trials
Trials by Disease
Information Classification: General
Q2
20
21
Overview of dealmaking for gene, cell, and RNA therapy companies
Information Classification: General
36 / Q2 2021
Alliance, acquisition, and financing in gene, cell, & RNA therapy
6 8
1712 10
49
40
72
5761
77
60
82
98
71
0
20
40
60
80
100
120
Q2 2020 Q3 2020 Q4 2020 Q1 2021 Q2 2021
Nu
mb
er o
f d
eals
Total number of deals by type, most recent five quarters
*Financing
Alliance
Acquisition
Source: Biomedtracker | Informa, July 2021 *Financings include public financings (IPOs and follow-ons) plus privately raised funding through venture rounds, debt offerings, or private investment in public equity
• The gene, cell, & RNA industry has seen a greater volume of alliances, plus a slight uptick in acquisitions in Q2 2021 vs one year ago
• Biggest decrease in Q2 2021 was seen in financings, with >20 fewer transactions done in Q2 than in Q1, and this was mainly attributable to fewer follow-on public offerings
Information Classification: General
37 / Q2 2021
Q2 2021 acquisitions in gene, cell, & RNA therapyDeal Date Deal Title Potential Deal Value (USD)
04/09/2021 Sanofi Acquires Tidal Therapeutics 470,000,000
04/20/2021 SparingVision Acquires GAMUT Therapeutics Undisclosed
05/04/2021Athenex Acquires Kuur Therapeutics to Expand Cell Therapy Development with Off-the-Shelf Engineered CAR-NKT Platform
185,000,000
05/17/2021 Charles River Laboratories to Acquire Vigene Biosciences to Enhance Gene Therapy Capabilities 350,000,000
06/01/2021 Auris Medical Acquires RNA Therapeutics Company Trasir, Changes Name and Strategic Focus Undisclosed
06/14/2021 Brooklyn ImmunoTherapeutics Executes Letter of Intent to Acquire Novellus Therapeutics 125,000,000
06/14/2021 Avalon GloboCare to Acquire SenlangBio in All Stock Transaction Undisclosed
06/22/2021uniQure to Acquire Corlieve Therapeutics and Advance its Gene Therapy Program to Treat Temporal Lobe Epilepsy
298,300,000
06/30/2021 Scopus BioPharma Expands Immunotherapy Pipeline with Acquisition of Olimmune Undisclosed
06/17/2021 Danaher Pays $9.6B for Aldevron, Manufacturer of High-Quality Plasmid DNA, mRNA, and Proteins 9,600,000,000
Source: Biomedtracker | Informa, July 2021
Information Classification: General
Q2
20
21
Start-up funding for gene, cell, & RNA therapy companies
Information Classification: General
39 / Q2 2021
Start-up financing for gene, cell, & RNA therapy companies
$1,043.7
$523.5
$744.8
$479.8
$1,445.3
11
1618
13
23
0
5
10
15
20
25
$-
$200.0
$400.0
$600.0
$800.0
$1,000.0
$1,200.0
$1,400.0
$1,600.0
Q2 2020 Q3 2020 Q4 2020 Q1 2021 Q2 2021
Nu
mb
er o
f fi
nan
cin
gs
Tota
l rai
sed
($
M)
Volume and dollar value of Series A and seed financings for gene, cell, & RNA therapy companies, most recent five quarters
Total raised ($M) Number of financingsSource: Biomedtracker | Informa, July 2021
• 23 companies raised seed or Series A financing in Q2 2021, reaching an aggregate $1.4 billion
• Nearly 75% (17/23) of biotechs are based in the US (mainly Massachusetts and California), the rest in Europe and Asia
• Top financing ($250M) was done by a new company created by Intellia, Cellex (and subsidiary GEMoaB), and Blackstone to develop allogeneic universal CAR-T therapies based on Intellia’s CRISPR/Cas9 platform and CEMoaB’s switchable, universal CAR T-cell platforms
Information Classification: General
40 / Q2 2021
Q2 2021 start-up financing for gene, cell, & RNA therapy companies (1/2)
Source: Biomedtracker | Informa, July 2021
Deal Date Deal Title Company Location Academic SourcePotential Deal
Value (USD)
04/07/2021Shoreline Biosciences Announces $43M Early Round To Advance Pipeline
United States, California, San Diego
University of California, San Diego 43,000,000
04/19/2021 Rejuvenate Bio Raises over $10M in Series A financing United States, California, San Diego
Harvard University: Harvard Medical School and Wyss Institute
10,000,000
04/20/2021 Code Biotherapeutics Launches with $10M in Seed FinancingUnited States, Pennsylvania, Hatfield
Undisclosed 10,000,000
04/29/2021 Capsida Biotherapeutics Debuts with $50M Series A RoundUnited States, California, Newbury Park
California Institute of Technology: Tianqiao and Chrissy Chen Institute for Neuroscience
50,000,000
04/30/2021 Treeline Biosciences Launches with $211M Series A RoundUnited States, Connecticut, Stamford
Undisclosed 211,669,568
05/05/2021 Isolere Bio Closes $7M in Seed FundingUnited States, North Carolina, Durham
Duke University 7,000,000
05/06/2021 Dyno Therapeutics Closes $100M Series A RoundUnited States, Massachusetts, Cambridge
Harvard University: Harvard Medical School and Wyss Institute
100,000,000
05/10/2021Gennao Bio Closes $40M Series A Financing to Develop Pipeline of Targeted Nucleic Acid Therapeutics
United States, New York, New York
Yale University 40,000,000
05/11/2021 Appia Bio Launches With $52M Series A FinancingUnited States, California, Los Angeles
University of California, Los Angeles 52,000,000
05/18/2021G2 Bio Companies Launch with $200M to Develop Transformative Genetic-Based Therapies
United States, Pennsylvania, Philadelphia
University of Pennsylvania: Gene Therapy Program 200,000,000
05/18/2021Vedere Bio II Launches with $77M Series A Financing to Develop Ocular Gene Therapies
United StatesUniversity of California, Berkeley; University of Pennsylvania: School of Veterinary Medicine
77,000,000
05/20/2021HAYA Therapeutics Completes CHF 18M Seed Financing To Advance Anti-fibrotic Therapies Targeting Long Non-coding RNAs
Switzerland, Vaud Lausanne University Hospital 19,985,500
05/27/2021 miRecule Inc. Closes $5.7M Seed FundingUnited States, Maryland, Gaithersburg
National Institutes of Health: BioHealth Innovation 5,700,000
Information Classification: General
41 / Q2 2021
Q2 2021 start-up financing for gene, cell, & RNA therapy companies (2/2)
Source: Biomedtracker | Informa, July 2021
Deal Date Deal Title Company Location Academic SourcePotential Deal
Value (USD)
06/09/2021Alcyone Therapeutics Launches with $23M to Advance Gene Therapies for CNS Disorders
United States, Massachusetts, Lowell
Nationwide Children's Hospital: Abigail Wexner Research Institute
23,000,000
06/15/2021 VectorY Raises $37.58M in Seed Financing Netherlands, Amsterdam Undisclosed 37,582,695
06/16/2021Mnemo Therapeutics Announces $90M Series A to Accelerate Next Generation Integrated CAR-T and Epigenetic Targeting Platform
France, Paris Institut Curie; Memorial Sloan Kettering Cancer Center 90,000,000
06/16/2021 Ochre Bio Closes $9.6M Seed Financing United Kingdom, Oxford Working in collaboration with Oxford University 9,600,000
06/18/2021Nuevocor Closes $24M Series A Financing to Advance Novel Gene Therapies for Cardiomyopathies
Singapore Agency for Science, Technology and Research (A*STAR) 24,000,000
6/22/2021Unnamed autologous/allogeneic CAR-T company launched with $250M from Blackstone Life Sciences, Intellia Therapeutics, and Cellex Cell Professionals
United States, Massachusetts, Cambridge
n/a – founded around pooled resources from GEMoaBGmbH (Cellex’s parent company) and Intellia
250,000,000
06/23/2021 Abata Therapeutics Launches with $95M Series A FundingUnited States, Massachusetts, Cambridge
Co-founders are from Harvard Medical School, Massachusetts Institute of Technology, and University Hospital of Zurich
95,000,000
06/23/2021 Vita Therapeutics Raises $32M in Series A FinancingUnited States, Maryland, Baltimore
Johns Hopkins University; Kennedy Krieger Institute 32,000,000
06/23/2021Strand Therapeutics Raises $52M via Oversubscribed Series A Round
United States, Massachusetts, Cambridge
MIT Synthetic Biology Center 52,000,000
06/24/2021OliX' subsidiary mCureX Therapeutics Raises ₩6.5B in Pre-Series A Investment
Korea (South) n/a – subsidiary of OliX Pharmaceuticals 5,738,860
Information Classification: General
42 / Q2 2021
Notable Q2 2021 start-up gene, cell, & RNA therapy companies
Source: Biomedtracker | Informa, July 2021; company websites
Company details Academic sourceFinancing
type/amount raisedLead investor(s) Therapy areas of interest
T-cell engineering EnfiniTplatform technology, including identification of antigens with greater tumor specificity and development of next-generation CAR-T therapies
Institut Curie; Memorial Sloan Kettering Cancer Center
Series A; $90M
Casdin Capital, SofinnovaPartners, and an undisclosed investor
Oncology
CapsidMap platform, which designs AAV vectors using artificial intelligence
Harvard University: Harvard Medical School and Wyss Institute
Series A/$100MAndreessen Horowitz
Liver, muscle, eye, central nervous system, lung, heart and kidney diseases
DiscoverHAYA platform to discover tissue and cell-specific long non-coding RNA drug candidates
Lausanne University Hospital
Seed/$20MBroadview Ventures
Fibrosis and conditions related to aging
Information Classification: General
44 / Q2 2021
Upcoming Catalysts
Therapy Generic Name Disease Catalyst Catalyst Date
Skysonaelivaldogene autotemcel, Lenti-D
Cerebral adrenoleukodystrophy
European Approval Decision
May 21 - Sep 30, 2021
Yescarta axicabtagene ciloleucel Marginal cell lymphoma FDA Approval Decision Jun 18 - Sep 30, 2021
Abecma idecabtagene vicleucel Multiple myelomaEuropean Approval Decision
Jun 24 - Aug 30, 2021
Below are noteworthy catalysts (forward looking events) that are expected in Q3 2021
Source: Biomedtracker | Informa, July 2021
Information Classification: General
46 / Q2 2021
Methodology: Sources and scope of therapies
Pipeline and trial data• Data derived from Citeline (Pharmaprojects and Trialtrove)• Therapeutic classes included in report categorizations:
• Gene therapies: Gene therapy; cellular therapy, chimeric antigen receptor; cellular therapy, T cell receptor; lytic virus
• Cell therapies: Cellular therapy, other; cellular therapy, stem cell; cellular therapy, tumor-infiltrating lymphocyte
• RNA therapies: Messenger RNA; oligonucleotide, non-antisense, non-RNAi; RNA interference; antisense therapy
Deal, financing, and catalyst data• Data derived from Biomedtracker• The following industry categorizations of deals are included: Gene
therapy, cell therapy; antisense, oligonucleotides
Sources for all data come from Informa Pharma Intelligence
Information Classification: General
47 / Q2 2021
Glossary of Key TermsTherapy Type Definitions
Gene therapy is the introduction, removal, or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease. For the purpose of this report, the following terms shall mean the following:
Gene therapy
Therapies containing an active ingredient synthesized following vector-mediated introduction of a genetic sequence into target cells in- or ex-vivo. Used to replace defective or missing genes (as in cystic fibrosis) as well as to introduce broadly acting genetic sequences for the treatment of multifactorial diseases (e.g. cancer). Direct administration of oligonucleotides without using vectors is covered separately in the antisense therapy class; RNA interference class; or oligonucleotide, non-antisense, non-RNAi class. Platform technologies for gene delivery are covered separately in the gene delivery vector class.
Cellular therapy, chimeric antigen receptor*Falls under gene therapy in this report
Cellular therapy consisting of T cells that have been modified to express a chimeric antigen receptor (CAR) – this is a cell surface receptor that gives the T cells the ability to target a specific protein and fight the targeted cells.
Cellular therapy, T cell receptor*Falls under gene therapy in this report
Cellular therapies whereby natural T-cells collected for the patient, are engineered to express artificial receptors (usually through viral transfections) that would target specific intracellular antigens (as peptides bound to proteins encoded by the major histocompatibility complex, MHC).
Lytic virus*Falls under gene therapy in this report
Therapies which have a replication-competent virus, that lyse pathogenic cells directly. These are normally genetically modified to render them harmless to normal tissues. Examples include oncolytic viruses which specifically attack cancer cells.
Information Classification: General
48 / Q2 2021
Glossary of Key TermsTherapy type definitions, cont.
Cell therapy includes the following therapeutic classes:
Cellular therapy, stem cellRegenerative therapy which promotes the repair response of injured tissue using stem cells (cells from which all other specialized cells would originate).
Cellular therapy, tumor infiltrating lymphocyteAdoptive cellular transfer of tumor resident T cells from tumor material, their expansion ex vivo, and transfer back into the same patient after a lymphodepleting preparative regimen.
Cellular therapy, otherCellular therapies that do not fall under the categories of cellular therapy, stem cell; cellular therapy, CAR; cellular therapy, TIL; cellular therapy, TCR; or the specific cellular therapy are unspecified.
Information Classification: General
49 / Q2 2021
Glossary of Key TermsTherapy type definitions, cont.
RNA therapy includes the following therapeutic classes:
Messenger RNATherapies that carry the desired mRNA code to overcome genetic mutations. The mRNA sequence will replace the defective mRNA in a patient and starts producing the desired protein.
Oligonucleotide, non-antisense, non-RNAi
Synthetic therapeutic oligonucleotides which operate by a mechanism other than antisense or RNA interference (RNAi). This includes ribozymes, aptamers, decoys, CpGs, and mismatched and immunostimulant oligonucleotides. Sequences delivered using vectors (gene therapy) are covered separately in “gene therapy.“ Antisense and RNAi oligonucleotides are covered separately in “antisense therapy" and "RNA interference,“ respectively.
RNA interference
Includes products which act therapeutically via an RNA interference (RNAi) mechanism, including small interfering RNAs (siRNAs). These may be synthetic oligonucleotides, or RNAi sequences may be expressed from a vector as a form of gene therapy (see “gene therapy" therapeutic class). In vivo, these sequences block the expression of a specific protein by forming an RNA-induced silencing complex, which then specifically binds to and degrades a complementary mRNA encoding the target protein. The use of RNAi purely as a drug discovery tool (e.g., in transgenic animal model production or in target validation) is not covered in this section.
Antisense therapy
Antisense compounds under development as potential therapeutics. These may be synthetic oligonucleotides, or antisense RNA may be expressed from a vector as a form of gene therapy. They may prevent the expression of a specific protein in vivo by binding to and inhibiting the action of mRNA, since they have a specific oligonucleotide sequence which is complementary to the DNA or RNA sequence which codes for the protein.
Information Classification: General
50 / Q2 2021
Glossary of Key TermsDevelopment status definitions
Pipeline Drugs that are in active development
Preclinical Not yet tested in humans
Phase I Early trials, usually in volunteers, safety, PK, PD
Phase II First efficacy trials in small numbers of patients
Phase III Large scale trials for registrational data
Pre-registration Filing for approval made to regulatory authorities
Approved Approval from relevant regulatory authorities for human use
Unspecified indications
Cancer, unspecified Indications for which the specific tumor type is not specified
Cancer, hematological, unspecified
Indications for which the specific hematological cancer is not specified
Cancer, solid, unspecified Indications for which the specific solid tumor is not specified
Deal type categories
Alliances Co-marketing, co-promotion, disease management, joint venture, manufacturing or supply, marketing-licensing, product or technology swap, product purchase, R+D and marketing-licensing, reverse licensing, trial collaborations
Financing Convertible debt, FOPO, IPO, nonconvertible debt, financing/other, private investment in public equity, private placement, royalty sale, special-purpose financing vehicle, spin-off
Acquisitions Buy-out, divestiture, spin-out, full acquisition, partial acquisition, reverse acquisition
Information Classification: General
51 / Q2 2021
Report Contributors
Ly Nguyen-Jatkoe, PhDExecutive Director, AmericasInforma Pharma Custom Intelligence
Amanda Micklus, MScSenior Pharma ConsultantInforma Pharma Custom Intelligence
David Barrett, JDCEOAmerican Society of Gene + Cell Therapy
Betsy Foss-Campbell, MADirector of Policy and AdvocacyAmerican Society of Gene + Cell Therapy
Alex Wendland, MSJDirector of CommunicationsAmerican Society of Gene + Cell Therapy
Doro Shin, MPHSenior Director, Content MarketingInforma Pharma Intelligence
Contact: David Barrett, JD at [email protected]
Contact:[email protected]