List of 88 Gene Therapies
Contributing Author Luke Williams, Sales and Marketing
Biotechnology is a rapidly expanding sector where game-changing innovation is a daily occurrence. Gene and cell therapies are of particular interest because of the steep technology curve driving the technology platform. The gene and cell therapy industry is characterized by numerous small, competitive, agile businesses; each with a unique toolset. The decreasing cost trend of these genetic technologies continually lowers the industry’s barriers to entry, allowing scores of creative entities to enter the market. As you read, consider the scale of the emergence of gene and cell therapies, and what the future holds for populations affected by the diseases addressed by these maturing therapeutics.
FDA Approved Gene Therapies
Onasemnogene Abeparvovec-Xioi
Disease: Spinal Muscular Atrophy
Indication: For the treatment of pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
AveXis - Private Company
- Notable Transactions: Based on the NAV AAV9 vector that was developed by RegenXBio. AveXis was acquired by Novartis $8.7B.
Methods: Utilizes NAV AAV9 to deliver functional copies of the SMN1 gene to neurons.
Package Insert: https://www.fda.gov/media/126109/download
Link to the clinical trial: NCT03306277
Link to company R&D pipeline: https://avexis.com/research-and-development.html
Axicabtagene Ciloleucel
Disease: Large B-cell Lymphoma
Indication: Treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.
Kite Pharma - Private Company
- Notable Transactions: Gilead acquired Kite Pharma for $12B
Methods: The patient sits for leukapheresis, and the product is sent to a lab. There, the T cells are genetically enhanced to attack cancerous cells and expanded.
Link to the clinical trial: NCT02348216
Link to company R&D pipeline: https://www.kitepharma.com/science-medicine/pipeline
Package Insert: https://www.fda.gov/media/108377/download
Voretigene neparvovec-rzyl
Disease: Retinal Dystrophy
Indication: Adeno-associated virus vector-based gene therapy indicated for the treatment of patients with biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).
Spark Therapeutics [NASDAQ:ONCE]
- IPO $161M Jan 2015
- Market Cap $4.38B Oct 2020
- Notable Transactions: Roche acquired Spark Therapeutics for $4.7B
Methods: A functional copy of RPE65 is introduced to patients via a subretinal injection of Adeno-associated viral vector solution.
Link to the clinical trial: NCT00999609
Link to company R&D pipeline: https://sparktx.com/scientific-platform-programs/
Package Insert: https://www.fda.gov/media/109906/download
https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna
Tisagenlecleucel
Disease: Large B-cell lymphoma
Indication: Patients up to 25 years of age with B-cell precursor acute lymphoblasticleukemia (ALL) that is refractory or in second or later relapse.
Novartis Pharmaceuticals [NYSE:NVS]
- Market Cap: $198.098B Oct 2020
- Notable Transactions:
Methods: Patient’s T cells are modified to recognize CD19 on B cells, expanded, and reintroduced to the patient to combat Large B-cell Lymphoma.
Link to the clinical trial: NCT04161118
Link to company R&D pipeline: https://www.novartis.com/our-science/novartis-global-pipeline
Package Insert: https://www.fda.gov/media/107296/download
(Approved in EU)
Disease: Beta-Thalassemia
Indication: Zynteglo is indicated for the treatment of patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
Bluebird Bio [NASDAQ:BLUE]
- IPO $116.1M Jun 2016
- Market Cap $3.713B Oct 2020
- Notable Transactions: Priced @ €1.58m / patient
Notes: Bluebird previously said it planned to file its FDA application for Zynteglo by year-end 2019, but it has yet to send the therapy to U.S. regulators.
Methods: Hematopoietic Stem Cells (HSCs) are collected from the patient, then treated with a lentiviral vector to introduce functional genes for beta-globulin. HSCs are then re-introduced to the patient.
Link to clinical trials: NCT01745120
Link to company R&D pipeline: https://www.bluebirdbio.com/our-science/pipeline
More information: https://www.ema.europa.eu/en/medicines/human/EPAR/zynteglo
Disease: X-linked Severe Combined Immunodeficiency (XSCID)
Indication: Not specified
Mustang Bio [NASDAQ:MBIO]
- IPO $32M May 2019
- Market Cap $190.088M Oct 2020
- Notable Transactions: Mustang Bio collaborated with St. Jude Children’s Hospital for the phase 1/2 clinical trial.
Methods: Inherited non-functional interleukin genes on the X chromosome are replaced by functional copies via lentiviral delivery.
Link to the clinical trial: NCTO1512888
Link to company R&D pipeline: https://www.mustangbio.com/pipeline/
(Approved in EU)
Disease: Adenosine Deaminase Deficiency (ADA-SCID)
Indication: Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
Orchard Therapeutics [NASDAQ:ORTX]
- IPO $255M Nov 2018
- Market Cap $471.19M Oct 2020
- Notable Transactions: Originally part of GlaxoSmithKline’s rare disease gene therapy unit. This asset, among others, was handed off to Orchard Therapeutics in 2018.
Package Insert: https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis
Methods: A sample of the patient’s bone marrow is taken, and the CD34+ cells are transduced with a retroviral vector in order to restore gene function before being reintroduced to the patient.
Link to the clinical trial: NCT03478670
Link to company R&D pipeline: https://www.orchard-tx.com/approach/pipeline/
brexucabtagene autoleucel
Disease: Mantle Cell Lymphoma
Indication: For the treatment of adult patients with relapsed/refractory mantle cell lymphoma (r/r MCL).
Kite Pharma - Private Company
- Notable Transactions: Gilead acquired Kite Pharma for $12B
Methods: Patient undergoes leukapheresis. Lymphocytes are expanded and transduced with genes that strengthen the immune response to CD19+ cells. Autologous anti-CD 19 CAR T cells are reintroduced to the patient intravenously.
Package Insert: https://www.fda.gov/media/140409/download
Link to the clinical trial: NCT02601313
Link to company R&D pipeline: https://www.kitepharma.com/science-medicine/pipeline
Phase 3
AMT-061
Etranacogene Dezaparvovec
Disease: Hemophilia B
Indication: For the treatment of patients with severe and moderately severe hemophilia B.
uniQure [NASDAQ:QURE]
- IPO $81.9M Feb 2014
- Market Cap $1.667B Oct 2020
- Notable Transactions: uniQure is a publicly traded company
Methods: Delivery of factor IX gene via adeno-associated virus serotype 5 (AAV5).
Link to the clinical trial: NCT03569891
Link to company R&D pipeline: http://uniqure.com/gene-therapy/pipeline.php
OTL-103
Disease: Wiskott Aldrich Syndrome
Indication: Unspecified
Orchard Therapeutics [NASDAQ:ORTX]
- IPO $255M Nov 2018
- Market Cap $471.19M Oct 2020
- Notable Transactions: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) collaborated on the phase 1/2 trial
Methods: Patient-derived Hematopoietic Stem Cells (HSCs) are transduced with functional copies of nonfunctional genes via a lentiviral vector.
Link to the clinical trial: NCT03837483
Link to company R&D pipeline: https://www.orchard-tx.com/approach/pipeline/
PTC-AADC
Disease: L-amino Acid Decarboxylase Deficiency
Indication: For patients, commonly pediatric, living with aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC Therapeutics [NASDAQ:PTCT]
- IPO $114.2M Jun 2013
- Market Cap $3.63B
- Notable Transactions: PTC Therapeutics has made deals to acquire Agilis Biotherapeutics and Censa Pharmaceuticals
Methods: A functional copy of the DDC gene is delivered via an adeno-associated viral vector by surgical injection.
Link to the clinical trial: NCT01395641
Link to company R&D pipeline: https://www.ptcbio.com/our-pipeline/portfolio-pipeline/
Lenti-D
ALD-104 and Starbeam ALD-102
Disease: Cerebral Adrenoleukodystrophy (CALD)
Indication: For male patients age 17 and younger afflicted by CALD
Bluebird Bio [NASDAQ:BLUE]
- IPO Jun 2016 $116.1M
- Market Cap $3.713B Oct 2020
- Notable Transactions: Publicly traded company
Methods: Replacement of the ABCD1 gene in CD34+ cells via a lentiviral vector.
Link to the clinical trial: NCT01896102
Link to company R&D pipeline: https://www.bluebirdbio.com/our-science/pipeline
VB-111
Ofranergene Obadenovec
Disease: Ovarian Cancer
Indication: For patients with ovarian cancer who have failed to respond to platinum-based chemotherapy.
Vascular Biogenics [NASDAQ:VBLT]
- IPO $34.4M Oct 2014
- Market Cap $56.039M Oct 2020
- Notable Transactions: Public Company
Methods: Two pronged approach to solid tumors - apoptotic genes are delivered to the cells in solid tumors via adeno-associated virus serotype 5.
Link to the clinical trial: NCT03398655
Link to company R&D pipeline: https://www.vblrx.com/cancer-programs/cancer-platform-technology/
QR-110
Sepofarsen
Disease: Leber’s congenital amaurosis (LCA)
Indication: For patients with bilateral, idiopathic Parkinson’s disease.
ProQR Therapeutics [NASDAQ:PRQR]
- IPO $98M Sept 2014
- Market Cap $242.691M Oct 2020
- Notable Transactions:
Methods: RNA-based therapeutic that repairs centrosomal protein 290kDA (CEP290).
Link to the clinical trial: NCT03913143
Link to company R&D pipeline: https://www.proqr.com/pipeline
EB-101
Disease: Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Indication: For patients with large, chronic, RDEB wounds.
Abeona Therapeutics [NASDAQ:ABEO]
- IPO $103.5M Dec 2019
- Market Cap $104.277M Oct 2020
- Notable Transactions: Phase 3 trial was postponed by the FDA in 2019 over concerns about the stability of EB-101 during transportation.
Methods: Delivers functional COL7A1 genes via an adeno-associated viral vector.
Link to the clinical trial: NCT04227106
Link to company R&D pipeline: https://www.abeonatherapeutics.com/science#pipeline
Lumevoq
GS010, lenadogene nolparvovec
Disease: Leber Hereditary Optic Neuropathy (LHON)
Indication: Not Specified
GenSight Biologics [EPA:SIGHT]
- IPO $44M Jul 2016
- Market Cap $145.7M Oct 2020
- Notable Transactions: Public Company
Methods: Using a retroviral vector, a functional copy of the type VII gene is transduced into keratinocytes.
Link to the clinical trial: NCT01263379
Link to company R&D pipeline: https://www.gensight-biologics.com/our-product-pipeline/
BIIB-111
Timrepigene Emparvovec
Disease: Choroideremia (CHM)
Indication: Unspecified
Biogen [NASDAQ:BIIB]
- IPO in 1983
- Market Cap: $45.238B Oct 2020
- Notable Transactions: Public company since 1983
Methods: Delivers a functional copy of the CHM gene via the AAV-REP2 vector.
Link to the clinical trial: NCT03496012
Link to company R&D pipeline: https://www.biogen.com/en_us/pipeline.html
BENEGENE-2
Fidanacogene Elaparvovec
Disease: Hemophilia B
Indication: For adult patients with Hemophilia B due to a nonfunctional coagulation factor IX gene.
Pfizer [NYSE:PFE]
- IPO $5.9M June 1942
- Market Cap $202.659B Oct 2020
- Notable Transactions: Transferred from Spark Therapeutics to Pfizer
Methods: Replacement of nonfunctional Factor IX genes using spark therapeutics’ AAV vector platform.
Link to the clinical trial: NCT03861273
Link to company R&D pipeline: https://www.pfizer.com/science/drug-product-pipeline
BIIB112
NSR-RPGR
Disease: X-linked Retinitis Pigmentosa (XLRP)
Indication: For patients with XLRP caused by RPGR gene mutation.
NightstaRx Ltd - Private Company
- Notable Transactions: a Biogen Company
Methods: Functional genes delivered via an AAV vector.
Link to the clinical trial: NCT03116113
Link to company R&D pipeline: https://www.biogen.com/en_us/pipeline.html
OTL-200
Disease: Metachromatic Leukodystrophy
Indication: For use in patients with Late Juvenile Metachromatic Leukodystrophy (MLD).
Orchard Therapeutics [NASDAQ:ORTX]
- IPO: $255M Nov 2018
- Market Cap: $471.19M Oct 2020
- Notable Transactions: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) collaborated on phase 1/2 trial.
Methods: Autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells (HSPC) transduced ex vivo with a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.
Link to the clinical trial: NCT04283227
Link to company R&D pipeline: https://www.orchard-tx.com/approach/pipeline/
Neovasculgen
Disease: Atherosclerotic Peripheral Arterial Disease (PAD), including Critical Limb Ischemia (CLI)
Indication: For patients with Atherosclerotic PAD, or CLI.
Human Stem Cells Institute
- Notable Transactions: Already approved in Russia, Human Stem Cells Institute has licensed the exclusive U.S. and Canadian rights for Neovasculgen to ArtGen, Inc.
Methods: Intramuscular transfer of a plasmid DNA encoding vascular endothelial growth factor (VEGF) 165 with cytomegalovirus promoter (CMV).
Link to the clinical trial: NCT03068585
Link to company R&D pipeline: N/A
Phases 1 and 2
Disease: HIV/AIDS
Indication: For subjects with chronic HIV disease and viremia suppressed by antiretroviral therapy
American Gene Technologies - Private Company
Methods: Autologous Gag-specific T cells are transduced with a lentiviral vector ex vivo. The modified HIV resistant T cell are then reintroduced to the patient intravenously.
Link to the clinical trial: NCT04561258
Link to company R&D pipeline: https://www.americangene.com/pipeline/
SRP-9001
Disease: Duchenne muscular dystrophy
Indication: Not Specified
Sarepta Therapeutics [NASDAQ:SRPT]
- IPO: $325M Jul 2017
- Market Cap: $11.451B Oct 2020
- Notable Transactions: Sarepta has licensed SRP-9001 to Roche
Methods: Delivery of our micro-dystrophin transgene from our AAVrh74 viral capsid and targeted muscle expression.
Link to the clinical trial: NCT03769116
Link to company R&D pipeline: https://www.sarepta.com/products-pipeline/pipeline
BMN-270
Valoctocogene Roxaparvovec
Disease: Hemophilia A
Indication: Not Specified
BioMarin Pharmaceuticals [NASDAQ:BMRN]
- IPO: $67.3M Jul 1998
- Market Cap: $13.701B Oct 2020
Methods: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII.
Link to the clinical trial: NCT03370913
Link to company R&D pipeline: https://www.biomarin.com/products/pipeline
CTX001
Disease: Sickle Cell Disease
Indication: For patients suffering from severe sickle cell disease (SCD)
CRISPR Therapeutics [NASDAQ:CRSP]
- IPO: $56M Oct 2016
- Market Cap: $6.854B Oct 2020
- Notable Transactions: Phase 1/2 spearheaded by Vertex Pharmaceuticals Inc.
Methods: Autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene.
Link to the clinical trial: NCT03745287
Link to company R&D pipeline: http://www.crisprtx.com/programs/pipeline
OXB-102
AXO-Lenti-PD
Disease: Parkinson's Disease
Indication: For patients with bilateral, idiopathic Parkinson’s disease
Axovant Gene Therapies [NASDAQ:AXGT]
- IPO: $315M Jun 2015
- Market Cap: $154.819M Oct 2020
- Notable Transactions: Axovant Gene Therapies is a wholly owned subsidiary of Oxford BioMedica
Methods: Delivery of biosynthetic enzymes for the biosynthesis of dopamine via a lentiviral vector.
Link to the clinical trial: NCT03720418
Link to company R&D pipeline: https://www.oxb.com/pipeline
HMI-102
Disease: Phenylketonuria (PKU)
Indication: For adult subjects with PKU due to PAH deficiency.
Homology Medicines [NASDAQ:FIXX]
- IPO: $144M Mar 2018
- Market Cap: $515.486M Oct 2020
Methods: Delivery of functional PAH via an adeno associated vector.
Link to the clinical trial: NCT03952156
Link to company R&D pipeline: https://www.homologymedicines.com/therapeutic-focus
RP-A501
Disease: Danon Disease
Indication: For male patients with Danon Disease (DD)
Rocket Pharmaceuticals Inc. [NASDAQ:RCKT]
- IPO: $90.6M Apr 2019
- Market Cap: $1.486B Oct 2020
Methods: Adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene.
Link to the clinical trial: NCT03882437
Link to company R&D pipeline: https://www.rocketpharma.com/pipeline/
LB-001
Disease: Methylmalonic Acidemia (MMA)
Indication: For patients with mutations in the MUT gene that cause MMA.
LogicBio Therapeutics [NASDAQ:LOGC]
- IPO: $70M Oct 2018
- Market Cap: $229.782M Oct 2020
Methods: Delivery of functional methylmalonyl-COA mutase (MMUT) gene via AAV.
Link to the clinical trial: Phase 1/2 trial announced recently
Link to company R&D pipeline: https://www.logicbio.com/pipeline/
Ad-RTS-hIL-12
Disease: Glioblastoma
Indication: For subjects with recurrent or progressive Glioblastoma
ZIOPHARM Oncology [NASDAQ:ZIOP]
- IPO: $90.4M Feb 2020
- Market Cap: $602.972M Oct 2020
Methods: Adenoviral vector encoding human pro-inflammatory cytokine interleukin-12.
Link to the clinical trial: NCT04006119
Link to company R&D pipeline: https://ziopharm.com/pipeline/
SGT-001
Disease: Duchenne muscular dystrophy (DMD)
Indication: For adolescents and children with Duchenne muscular dystrophy (DMD)
Solid Biosciences, LLC [NASDAQ:SLDB]
- IPO: $133.7M Jan 2018
- Market Cap: $201.465M Oct 2020
Methods: AAV delivers of a gene encoding an smaller artificial version of the DMD gene.
Link to the clinical trial: NCT03368742
Link to company R&D pipeline: https://www.solidbio.com/research-development/pipeline-programs/pipeline
B-VEC
KB103
Disease: Dystrophic epidermolysis bullosa (DEB)
Indication: For adult patients with recessive DEB
Krystal Biotech [NASDAQ:KRYS]
- IPO: $45.54M Sept 2017
- Market Cap: $943.68M Oct 2020
Methods: A modified herpes simplex vector delivers a functional COL7A1 gene,
Link to the clinical trial: NCT04491604
Link to company R&D pipeline: https://www.krystalbio.com/focus/pipeline/
SRP-9003
MYO-101
Disease: Limb-girdle Muscular Dystrophy
Indication: For the treatment of limb-girdle muscular dystrophy (LGMD) type 2E.
Sarepta Therapeutics [NASDAQ:SRPT]
- IPO: $325M Jul 2017
- Market Cap: $11.451B Oct 2020
- Notable Transactions: Originally developed by researchers at the Nationwide Children’s Hospital Center for Gene Therapy, and was handed off to Sarepta Therapeutics for clinical trials.
Methods: Delivery of functional Beta-sarcoglycan (SG) genes via AAV.
Link to the clinical trial: NCT03652259
Link to company R&D pipeline: https://www.sarepta.com/products-pipeline/pipeline
RG6357
SPK-8011
Disease: Hemophilia A
Indication: For patients with Hemophilia A based on defective VIII genes.
Roche [OTCMKTS: RHHBY]
- IPO in 1919
- Market Cap: $292.134B Oct 2020
- Notable Transactions: Asset is being tested in clinical trials by Spark Therapeutics.
Methods: Delivery of functional Factor VIII genes via AAV.
Link to the clinical trial: NCT03003533
Link to company R&D pipeline: https://www.roche.com/research_and_development/who_we_are_how_we_work/pipeline.htm
RP-L102
Disease: Fanconi Anemia Subtype A (FA-A)
Indication: For pediatric patients with Fanconi anemia subtype A (FA-A)
Rocket Pharmaceuticals Inc. [NASDAQ:RCKT]
- IPO: $90.6M Apr 2019
- Market Cap: $1.486B Oct 2020
Methods: Autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene.
Link to the clinical trial: NCT03814408
Link to company R&D pipeline: https://www.rocketpharma.com/pipeline/
AVR-RD-01
Disease: Fabry Disease
Indication: For patients with mutations that cause the GLA gene to be defective
AVROBIO [NASDAQ:AVRO]
- IPO: $100M Feb 2020
- Market Cap: $520.765M Oct 2020
Methods: Autologous CD34+ cell-enriched fraction that contains cells transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence.
Link to the clinical trial: NCT03454893
Link to company R&D pipeline: https://www.avrobio.com/our-pipeline
MYO-201
SRP-6004
Disease: Duchenne Muscular Dystrophy
Indication: For patients with Duchenne Muscular Dystrophy
Sarepta Therapeutics [NASDAQ:SRPT]
- IPO: $325M Jul 2017
- Market Cap: $11.451B Oct 2020
- Notable Transactions: Sarepta Therapeutics acquired Myonexus
Methods: A gene therapy for LGMD2B designed to permanently enable muscle cells to produce dysferlin protein. Simultaneous intravenous administration of two vectors, each containing approximately one-half of the gene; the two halves self-assemble into a complete gene with a promoter that only expresses in muscle cells.
Link to the clinical trial: Link unavailable
Link to company R&D pipeline: https://www.sarepta.com/products-pipeline/pipeline
RGX-314
Disease: Wet Macular Degeneration
Indication: For wet AMD, diabetic retinopathy and other additional chronic retinal conditions treated with anti-VEGF
RegenXBio [NASDAQ:RGNX]
- IPO: $201.8M Aug 2018
- Market Cap: $1.049B Oct 2020
Methods: Delivery of the sequence for a soluble anti-VEGF protein via AAV.
Link to the clinical trial: NCT03066258
Link to company R&D pipeline: https://www.regenxbio.com/therapeutic-programs/
AAV-GAD
Disease: Parkinson's Disease
Indication: For patients with Parkinson’s disease caused by a defective GAD gene
MeiraGTx [NASDAQ:MGTX]
- IPO: $75M Jun 2018
- Market Cap: $542.136M Oct 2020
- Notable Transactions: Utilizes AAV vector from RegenXBio
Methods: Delivery of the GAD gene via bilateral surgical infusion of AAV-GAD into the subthalamic nucleus.
Link to the clinical trial: NCT00643890
Link to company R&D pipeline: https://meiragtx.com/research-development/pipeline/
OTL-203
Disease: Mucopolysaccharidosis type I (MPS-I)
Indication: For pediatric patients with bilateral, idiopathic Parkinson’s disease
Orchard Therapeutics [NASDAQ:ORTX]
- IPO: $255M Nov 2018
- Market Cap: $471.19M Oct 2020
Methods: Autologous gene that uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells.
Link to the clinical trial: Overseas Trial - https://www.globenewswire.com/news-release/2020/05/15/2034087/0/en/Orchard-Therapeutics-Presents-New-Interim-Data-from-OTL-203-Proof-of-concept-Study-for-MPS-I.html
Link to company R&D pipeline: https://www.orchard-tx.com/approach/pipeline/
MYO-102
SRP-9004
Disease: Muscular Dystrophy
Indication: For patients with treat limb-girdle muscular dystrophy type 2D (LGMD2D)
Sarepta Therapeutics [NASDAQ:SRPT]
- IPO: $325M Jul 2017
- Market Cap: $11.451B Oct 2020
- Notable Transactions: Sarepta Therapeutics acquired Myonexus
Methods: Delivery of alpha-sarcoglycan genes via AAV.
Link to the clinical trial: NCT01976091
Link to company R&D pipeline: https://www.sarepta.com/products-pipeline/pipeline
DTX401
Disease: Glycogen Storage Disease
Indication: For patients with Glycogen Storage Disease Type 1a (GSDIa)
Ultragenyx Pharmaceuticals [NASDAQ:RARE]
- IPO: $126.4M Feb 2014
- Market Cap: $5.708B Oct 2020
- Notable Transactions:
Methods: Delivery of Glucose-6- Phosphatase (G6Pase) via AAV,
Link to the clinical trial: NCT03517085
Link to company R&D pipeline: https://www.ultragenyx.com/pipeline/
VY-AADC
VY-AADC01, VY-AADC02
Disease: Parkinson's Disease
Indication: For patients with advanced Parkinson's disease, whose treatment response has started fluctuating.
Neurocrine Biosciences [NASDAQ:NBIX]
- IPO: $34.2M Jul 1999
- Market Cap: $9.499B Oct 2020
- Notable Transactions: developed by Voyager Therapeutics and evaluated clinically in partnership with Neurocrine Biosciences.
Methods: Delivery of the hAADC gene via AAV2.
Link to the clinical trial: NCT01973543
Link to company R&D pipeline: https://www.voyagertherapeutics.com/our-approach-programs/clinical-trials/
AAV-AQP1
AAV-Aquaporin-1
Disease: Sjogren's Syndrome
Indication: For patients with defective or inadequate aquaporin expression causing Sjogren's Syndrome.
MeiraGTx [NASDAQ:MGTX]
- IPO: $75M Jun 2018
- Market Cap: $542.136M Oct 2020
- Notable Transactions: Utilizes AAV vector from RegenXBio
Methods: Transferring the human aquaporin-1 (hAQP1) cDNA gene via AAV.
Link to the clinical trial: NCT02446249
Link to company R&D pipeline: https://meiragtx.com/research-development/pipeline/
EDIT-101
AGN-151587
Disease: Leber congenital amaurosis 10 (LCA10)
Indication: For patients with bilateral, idiopathic Parkinson’s disease
Editas Medicine [NASDAQ:EDIT]
- IPO: $94.4M Feb 2016
- Market Cap: $1.933B Oct 2020
- Notable Transactions: Allergan and Editas Medicine collaborated in the phase 1/2 trial as one entity, the Allergan-Editas Eye Program.
Methods: AGN-151587 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.
Link to the clinical trial: NCT03872479
Link to company R&D pipeline: https://www.editasmedicine.com/gene-editing-pipeline/
DTX301
Disease: Ornithine Transcarbamylase (OTC) Deficiency
Indication: For adult patients OTC deficiency
Ultragenyx Pharmaceuticals [NASDAQ:RARE]
- IPO: $126.4M Feb 2014
- Market Cap: $5.708B Oct 2020
- Notable Transactions:
Methods: AAV8-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC).
Link to the clinical trial: NCT02991144
Link to company R&D pipeline: https://www.ultragenyx.com/pipeline/
ADVM-022
Disease: Wet Macular Degeneration (AMD)
Indication: For adult patients with wet age-related macular degeneration
Adverum Biotechnologies [NASDAQ:ADVM]
- IPO: $216.8M Aug 2020
- Market Cap: $1.07B Oct 2020
Methods: Gene therapy delivering a coding sequence for aflibercept via AAV.7m8.
Link to the clinical trial: NCT03748784
Link to company R&D pipeline: https://adverum.com/pipeline/
TAVO
tavokinogene telseplasmid
Disease: Multiple Cancer Conditions
Indication: For patients with cancer conditions that could be aided by increased IL-12 production
OncoSec Medical [NASDAQ:ONCS]
- IPO: $7.75M Mar 2012
- Market Cap: $85.532M Oct 2020
Methods: A DNA plasmid that encodes genes for both the p35 and p40 subunits of the heterodimeric human interleukin 12 (hIL-12) protein.
Link to the clinical trial: NCT03132675
Link to company R&D pipeline: https://oncosec.com/pipeline/
RGX-111
Disease: Mucopolysaccharidosis Type I (MPS I)
Indication: For pediatric patients with MPS I
RegenXBio [NASDAQ:RGNX]
- IPO: $201.8M Aug 2018
- Market Cap: $1.049B Oct 2020
Methods: Gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system.
Link to the clinical trial: NCT03580083
Link to company R&D pipeline: https://www.regenxbio.com/therapeutic-programs/
OXB-201
RetinoStat
Disease: Wet Macular Degeneration (AMD)
Indication: For patients with advanced wet age-related macular degeneration (AMD)
Oxford BioMedica [LON:OXB]
- IPO in 1996
- Market Cap: $679.854M Oct 2020
Methods: Subretinal injection of a lentiviral vector carrying endostatin and angiostatin.
Link to the clinical trial: NCT01301443
Link to company R&D pipeline: https://oxb.com/pipeline
AT132
Disease: X-linked Myotubular Myopathy (XLMTM)
Indication: For infants with XLMTM
Axovant Gene Therapies [NASDAQ:AXGT]
- IPO: $315M Jun 2015
- Market Cap: $154.819M Oct 2020
- Notable Transactions: Axovant Gene Therapies is an Astellas Pharma company.
Methods: AAV8 vector containing a functional copy of the human MTM1 (hMTM1) gene.
Link to the clinical trial: NCT03199469
Link to company R&D pipeline: https://www.audentestx.com/x-linked-myotubular-myopathy/
AVXS-201
Disease: Rett Syndrome
Indication: For pediatric patients, primarily female, with X-linked Rett Syndrome
Novartis Pharmaceuticals [NYSE:NVS]
- Market Cap: $198.098B Oct 2020
- Notable Transactions: AveXis, the developer of AVXS-201 was acquired by Novartis
Methods: AAV-mediated delivery of a functional MECP2 gene copy.
Link to the clinical trial: Clinical trial scheduled but interrupted due to FDA data concerns
Link to company R&D pipeline: https://www.novartis.com/our-science/novartis-global-pipeline
RP-L201
Disease: Leukocyte Adhesion Defect - Type I (LAD-I)
Indication: For pediatric patients with LAD-I
Rocket Pharmaceuticals Inc. [NASDAQ:RKT]
- IPO: $90.6M Apr 2019
- Market Cap: $1.486B Oct 2020
Methods: Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene.
Link to the clinical trial: NCT03812263
Link to company R&D pipeline: https://www.rocketpharma.com/pipeline/
ABO-102
Disease: Sanfilippo syndrome type A
Indication: For pediatric patients with Sanfilippo syndrome type A
Abeona Therapeutics [NASDAQ:ABEO]
- IPO $103.5M Dec 2019
- Market Cap $104.277M Oct 2020
Methods: AAV-mediated delivery of SGSH gene.
Link to the clinical trial: NCT04088734
Link to company R&D pipeline: https://www.abeonatherapeutics.com/science#pipeline
ST-920
Disease: Fabry Disease
Indication: For patients with Fabry Disease
Sangamo Therapeutics [NASDAQ:SGMO]
- IPO: $145.5M Apr 2019
- Market Cap: $1.435B Oct 2020
Methods: Recombinant AAV2/6 vector encoding the cDNA for human a-Gal A.
Link to the clinical trial: NCT04046224
Link to company R&D pipeline: https://www.sangamo.com/pipeline
OTL-300
GSK2696277
Disease: Transfusion-Dependent Beta-Thalassemia (TDT)
Indication: Unspecified
Orchard Therapeutics [NASDAQ:ORTX]
- IPO: $255M Nov 2018
- Market Cap: $471.19M Oct 2020
- Notable Transactions: Formerly known as GSK2696277, this therapeutic was previously developed by GlaxoSmithKline.
Methods: Autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene.
Link to the clinical trial: NCT03275051
Link to company R&D pipeline: https://www.orchard-tx.com/approach/pipeline/
AT-GTX-501
scAVV9.CB.CLN6
Disease: vLINCL6 Disease
Indication: For patients with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene
Amicus Therapeutics [NASDAQ:FOLD]
- IPO: $75M May 2007
- Market Cap: $3.821B Oct 2020
Methods: Delivery of the CLN6 Gene by Self-Complementary AAV9.
Link to the clinical trial: NCT02725580
Link to company R&D pipeline: https://www.amicusrx.com/programs-pipeline/
AT-GTX-502
scAAV9.P546.CLN3
Disease: Batten disease.
Indication: For pediatric patients with CLN3 Batten disease.
Amicus Therapeutics [NASDAQ:FOLD]
- IPO: $75M May 2007
- Market Cap: $3.821B Oct 2020
Methods: Delivering the CLN3 Gene by Self-Complementary AAV9.
Link to the clinical trial: NCT03770572
Link to company R&D pipeline: https://www.amicusrx.com/programs-pipeline/
ACHM-CNGB3
rAAV2tYF-PR1.7-hCNGB3
Disease: Achromatopsia
Indication: For patients with Achromatopsia due to a mutation in the CNGB3 gene
Applied Genetic Technologies [NASDAQ:AGTC]
- IPO: $51.8M Apr 2014
- Market Cap: $141.443M Oct 2020
Methods: Subretinal injection of AAV carrying the CNGB3 gene.
Link to the clinical trial: NCT02599922
Link to company R&D pipeline: https://agtc.com/programs/
AVR-RD-02
Disease: Type 1 Gaucher disease
Indication: For postpubertal patients with a confirmed diagnosis of Type 1 Gaucher disease
AVROBIO [NASDAQ:AVRO]
- IPO: $100M Feb 2020
- Market Cap: $520.765M Oct 2020
Methods: Autologous CD34+ enriched hematopoietic stem cells that have been genetically modified with a lentiviral vector containing functional GCase genes.
Link to the clinical trial: NCT04145037
Link to company R&D pipeline: https://www.avrobio.com/our-pipeline
AGTC-402
Disease: Achromatopsia (ACHM)
Indication: For patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene
Applied Genetic Technologies [NASDAQ:AGTC]
- IPO: $51.8M Apr 2014
- Market Cap: $141.443M Oct 2020
Methods: Subretinal injection of AAV carrying the CNGA3 gene.
Link to the clinical trial: NCT02935517
Link to company R&D pipeline: https://agtc.com/programs/
AXO-AAV-GM1
Disease: GM1 Gangliosidosis
Indication: For patients with GM1 gangliosidosis due to nonfunctional β-galactosidase (β-gal) from a defective GLB1 gene
Axovant Gene Therapies [NASDAQ:AXGT]
- IPO: $315M Jun 2015
- Market Cap: $154.819M Oct 2020
Methods: Intravenous administration of AAV containing a normal copy of the GLB1 gene to the CNS.
Link to the clinical trial: NCT03952637
Link to company R&D pipeline: https://www.axovant.com/pipeline-overview
AGTC-501
Disease: X-linked retinitis pigmentosa (XLRP)
Indication: For patients with (XLRP) caused by mutations in the RPGR gene
Applied Genetic Technologies [NASDAQ:AGTC]
- IPO: $51.8M Apr 2014
- Market Cap: $141.443M Oct 2020
Methods: AAV carrying a functional RPGR gene.
Link to the clinical trial: NCT03316560
Link to company R&D pipeline: https://agtc.com/programs/
ABO-101
Disease: Sanfilippo Type B
Indication: For pediatric patients with Sanfilippo syndrome type A
Abeona Therapeutics [NASDAQ:ABEO]
- IPO $103.5M Dec 2019
- Market Cap $104.277M Oct 2020
Methods: AAV9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) delivered intravenously.
Link to the clinical trial: NCT03315182
Link to company R&D pipeline: https://www.abeonatherapeutics.com/science#pipeline
SB-318
Disease: Mucopolysaccharidosis Type I (MPS I)
Indication: For patients with MPS I
Sangamo Therapeutics [NASDAQ:SGMO]
- IPO: $145.5M Apr 2019
- Market Cap: $1.435B Oct 2020
Methods: Intravenously delivered Zinc Finger Nuclease (ZFN) that inserts a functional copy of the α-L-iduronidase (IDUA) gene.
Link to the clinical trial: NCT02702115
Link to company R&D pipeline: https://www.sangamo.com/pipeline
AXO-AAV-GM2
Disease: Tay-Sachs and Sandhoff diseases
Indication: For patients with GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) due to loss of function in β-hexosaminidase A (Hex A) from defective HEXA and HEXB genes.
Axovant Gene Therapies [NASDAQ:AXGT]
- IPO: $315M Jun 2015
- Market Cap: $154.819M Oct 2020
- Notable Transactions: Axovant Gene Therapies is a wholly owned subsidiary of Oxford BioMedica.
Methods: AAV carrying functional genes that encode β-Hexosaminidase A enzyme activity in central nervous tissue.
Link to the clinical trial: Human trials overseas - http://investors.axovant.com/news-releases/news-release-details/axovant-presents-first-evidence-clinical-stabilization-tay-sachs
Link to company R&D pipeline: https://www.axovant.com/pipeline-overview
AAV-RPE65
Disease: Retinal Dystrophy
Indication: For patients with retinal dystrophy associated with disease-causing variants in the RPE65 gene
MeiraGTx [NASDAQ:MGTX]
- IPO: $75M Jun 2018
- Market Cap: $542.136M Oct 2020
Methods: AAV encoding human RPE65 genes.
Link to the clinical trial: NCT02781480
Link to company R&D pipeline: https://meiragtx.com/research-development/pipeline/
RG6367
AAV2-hCGM, SPK-7001, spk-chm
Disease: Blindness
Indication: For patients with Choroideremia
Roche [OTCMKTS: RHHBY]
- IPO in 1919
- Market Cap: $292.134B Oct 2020
- Notable Transactions: Roche’s acquired this therapeutic along with Spark Therapeutics Inc.
Methods: Transduction of the CHM gene via AAV.
Link to the clinical trial: NCT02341807
Link to company R&D pipeline: https://www.roche.com/research_and_development/who_we_are_how_we_work/pipeline.htm
GEN-1
Disease: Ovarian Cancer
Indication: For newly diagnosed patients with Advanced Epithelial Ovarian, Fallopian Tube or Primary Peritoneal Cancer
Celsion [NASDAQ:CLSN]
- IPO: $9.3M Jun 2020
- Market Cap: $23.656M Oct 2020
Methods: Intravenous delivery of IL-12 Plasmid Formulated with PEG-PEI-Cholesterol Lipopolymer.
Link to the clinical trial: NCT03393884
Link to company R&D pipeline: https://celsion.com/pipeline/
AVRO-RD-04
Disease: Cystinosis
Indication: For adult and adolescent patients with Cystinosis
AVROBIO [NASDAQ:AVRO]
- IPO: $100M Feb 2020
- Market Cap: $520.765M Oct 2020
Methods: Autologous Human CD34+ Cells From Mobilized Peripheral Blood Stem Cells of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS Lentiviral Vector.
Link to the clinical trial: NCT03897361
Link to company R&D pipeline: https://www.avrobio.com/our-pipeline
QR-313
QRX-313
Disease: Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Indication: For patients with RDEB due to mutation(s) in exon 73 of the COL7A1 gene.
Wings Therapeutics - Private company
- Notable Transactions: Wings Therapeutics is a private spin-off company of ProQR Therapeutics
Methods: Topically applied QR-313 induces mRNA skipping in exon 73 of the COL7A1 gene.
Link to the clinical trial: NCT03605069
Link to company: https://www.wings-tx.com/
LMI070
Branaplam, NVS-SM1
Disease: Spinal Muscular Atrophy Type 1 (SMA 1)
Indication: For babies newly diagnosed with SMA 1
Novartis Pharmaceuticals [NYSE:NVS]
- Market Cap: $198.098B Oct 2020
Methods: Small molecule that induces expression of SMN protein from the SMN2 gene.
Link to the clinical trial: NCT02268552
Link to company R&D pipeline: https://www.novartis.com/our-science/novartis-global-pipeline
RGX-121
Disease: Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome
Indication: For pediatric patients with confirmed MPS II
RegenXBio [NASDAQ:RGNX]
- IPO: $201.8M Aug 2018
- Market Cap: $1.049B Oct 2020
Methods: Delivery of a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system via AAV.
Link to the clinical trial: NCT03566043
Link to company R&D pipeline: https://www.regenxbio.com/therapeutic-programs/
RGX-501
Disease: Homozygous Familial Hypercholesterolemia (HoFH)
Indication: For patients with HoFH due to dysfunctional expression of the human low-density lipoprotein receptor (LDLR) gene in liver cells.
RegenXBio [NASDAQ:RGNX]
- IPO: $201.8M Aug 2018
- Market Cap: $1.049B Oct 2020
Methods: Functional replacement of the defective LDLR via AAV-based liver-directed gene therapy.
Link to the clinical trial: NCT04080050
Link to company R&D pipeline: https://www.regenxbio.com/therapeutic-programs/
RG6358
SPK-8016
Disease: Hemophilia A with inhibitors to factor VIII
Indication: For individuals with Hemophilia A and FVIII inhibitors
Roche [OTCMKTS: RHHBY]
- IPO in 1919
- Market Cap: $292.134B Oct 2020
- Notable Transactions: Developed by Spark Therapeutics
Methods: Transduction of genes to restore Factor VIII functionality via AAV.
Link to the clinical trial: NCT03734588
Link to company R&D pipeline: https://www.roche.com/research_and_development/who_we_are_how_we_work/pipeline.htm
INXN-4001
Disease: Heart Failure
Indication: For patients in danger of heart failure, or who have survived a previous cardiac episode, due to faulty expression of human S100A1, SDF-1α, and VEGF165 gene products.
Triple-Gene LLC - Private Company
- Notable Transactions: Triple-Gene LLC is a majority owned subsidiary of Intrexon Corporation
Methods: Retrograde coronary sinus infusion of triple effector plasmid.
Link to the clinical trial: NCT03409627
Link to company R&D pipeline: https://precigen.com/pipeline/
RP-L401
Disease: Osteopetrosis
Indication: For infants afflicted by Infantile Malignant Osteopetrosis (IMO)
Rocket Pharmaceuticals Inc. [NASDAQ:RCKT]
- IPO: $90.6M Apr 2019
- Market Cap: $1.486B Oct 2020
Methods: Autologous CD34+ Cells With a Lentiviral Vector Encoding the TCIRG1 Gene.
Link to the clinical trial: NCT04525352
Link to company R&D pipeline: https://www.rocketpharma.com/pipeline/
Preclinical
MYO-301
SRP-9006
Disease: Muscular Dystrophy
Indication: For patients with limb-girdle muscular dystrophy type 2L
Sarepta Therapeutics [NASDAQ:SRPT]
- IPO: $325M Jul 2017
- Market Cap: $11.451B Oct 2020
- Notable Transactions: Sarepta Therapeutics acquired Myonexus
Methods: Unspecified
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.sarepta.com/products-pipeline/pipeline
HMI-103
Disease: Phenylketonuria (PKU)
Indication: For pediatric patients with PKU. Children’s livers are rapidly dividing so inserting a functional copy of the PAH gene early is the best approach for a potential cure in this patient population.
Homology Medicines [NASDAQ:FIXX]
- IPO: $144M Mar 2018
- Market Cap: $515.486M Oct 2020
Methods: AAVHSC carrying a functional copy of the PAH gene.
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.homologymedicines.com/therapeutic-focus
EDIT-301
Disease: Sickle Cell Diseases
Indication: For patients with mutations at the HBG1/2 promoter region, leading to faulty hemoglobin expression
Editas Medicine [NASDAQ:EDIT]
- IPO: $94.4M Feb 2016
- Market Cap: $1.933B Oct 2020
- Notable Transactions: Allergan and Editas Medicine collaborated in the phase 1/2 trial as one entity, the Allergan-Editas Eye Program.
Methods: Autologous CD34+ hematopoietic stem cells from sickle patients that are edited at the HBG1/2 promoter in the beta-globin locus using Cas12a (also known as Cpf1).
Link to the clinical trial: Preclinical - https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-presents-pre-clinical-data-study-edit-301-sickle
Link to company R&D pipeline: https://www.editasmedicine.com/gene-editing-pipeline/
LB-101
Disease: Hemophilia B
Indication: For patients with Hemophilia B due to a dysfunctional human coagulation factor IX gene
LogicBio Therapeutics [NASDAQ:LOGC]
- IPO: $70M Oct 2018
- Market Cap: $229.782M Oct 2020
Methods: AAV carrying Factor IX genes using LogicBio’s GeneRide technology.
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.logicbio.com/pipeline/
AAT Research Program
Disease: Alpha-1 Antitrypsin (AAT) Deficiency
Indication: For patients with AAT due to an abnormal human alpha-1 antitrypsin gene
Intellia [NASDAQ:NTLA]
- IPO: $112.9M May 2016
- Market Cap: $1.397B Oct 2020
Methods: Consecutive dosing of two distinct lipid nanoparticle (LNP) formulations, in adult mice, achieves two targeted genome editing events, resulting in knocking out the faulty gene and restoring therapeutic levels of normal alpha-1 antitrypsin protein (hAAT).
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.intelliatx.com/pipeline-2/
HMI-202
Disease: Metachromatic Leukodystrophy (MLD)
Indication: For infants that develop the late infantile form of MLD
Homology Medicines [NASDAQ:FIXX]
- IPO: $144M Mar 2018
- Market Cap: $515.486M Oct 2020
Methods: Delivery of a functional copy of the ARSA gene via AAV.
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.homologymedicines.com/therapeutic-focus
AVR-RD-03
Disease: Pompe disease
Indication: Preclinical. Intended for patients with afflicted by Pompe disease
AVROBIO [NASDAQ:AVRO]
- IPO: $100M Feb 2020
- Market Cap: $520.765M Oct 2020
Methods: Transduction of the GAA gene via a lentiviral vector.
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.avrobio.com/our-pipeline
MYO-103
Disease: Muscular Dystrophy Type 2C (LGMD2C)
Indication: For patients with LGMD2C
Sarepta Therapeutics [NASDAQ:SRPT]
- IPO: $325M Jul 2017
- Market Cap: $11.451B Oct 2020
- Notable Transactions: Sarepta Therapeutics acquired Myonexus
Methods: Delivery of alpha-sarcoglycan genes via AAV.
Link to the clinical trial: Preclinical
BBP-631
Disease: Congenital Adrenal Hyperplasia
Indication: For patients with Congenital Adrenal Hyperplasia (CAH)
BridgeBio Pharma [NASDAQ:BBIO]
- IPO: Jun 2019 $348.5M
- Market Cap: $4.932 Oct 2020
Methods: Delivery of 21-hydroxylase gene via AAV5.
Link to the clinical trial: Pre-clinical
Link to company R&D pipeline: https://bridgebio.com/pipeline
RTX-321
RTX-321 aAPC (HPV+)
Disease: HPV 16-positive cancers
Indication: For patients with HPV 16 positive tumors
Rubius Therapeutics [NASDAQ:RUBY]
- IPO: $241M Jul 2018
- Market Cap: $434.528M Oct 2020
Methods: Induces a tumor-specific immune response by expanding antigen-specific T cells. RTX-321 expresses an HPV peptide antigen bound to MHC I, 4-1BBL and IL-12 on the cell surface to mimic human T cell-APC interactions.
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.rubiustx.com/our-pipeline/
QR-411
Disease: Usher syndrome
Indication: For patients with vision loss due to Usher syndrome 2A PE40 caused by ineffective expression of the USH2A gene
ProQR Therapeutics [NASDAQ:PRQR]
- IPO $98M Sept 2014
- Market Cap $242.691M Oct 2020
Methods: RNA therapy that aims to prevent vision loss or restore vision in retinitis pigmentosa (RP) and Usher syndrome type 2 due to the PE40 mutation in the USH2A gene,
Link to the clinical trial: Preclinical
Link to company R&D pipeline: https://www.proqr.com/pipeline
