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Therapeutics to Present New Preclinical STAR-0215 Data at the European Academy of Allergy and Clinical Immunology 2022 Hybrid Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"orgOrder":0,"company":"SwanBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SwanBio Presents Design of Innovative Natural History Study Aimed to Evolve Understanding of Adrenomyeloneuropathy and Inform Future Treatments","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SwanBio Therapeutics"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astria Therapeutics Presents New Preclinical Data Showing Differentiated Profile of STAR-0215, in Development for Treatment of Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jnana Therapeutics to Present Preclinical Data for its PKU Program at the 2022 National PKU Alliance (NPKUA) Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Jnana Therapeutics"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cortexyme Successfully Completes Acquisition of Novosteo","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Quince Therapeutics"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Announces Optimized, In Vivo Gene Therapy Candidate for the Treatment of Metachromatic Leukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Verve Therapeutics Highlights Preclinical Data Supporting Nomination of VERVE-201 ANGPTL3 Product Candidate at the European Society of Cardiology 2022 Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Verve Therapeutics"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BrainVectis, a subsidiary of AskBio, Receives Clearance to Conduct Phase I\/II clinical trial in France for Its Novel Gene Therapy for Early-stage Huntington's Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"AskBio"},{"orgOrder":0,"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus to Present New Data on Its Amyloidosis Portfolio at the 2022 International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"orgOrder":0,"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"iECURE Receives FDA Rare Pediatric Disease Designation for GTP-506, an Investigational Gene Editing Product Candidate for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"iECURE"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Paragon Biosciences","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Castle Creek Biosciences Announces Publication of Preclinical Study for Potential In Vivo Gene Therapy in Nature Communications","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Castle Creek Biosciences"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Cure AP-4","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Charles River Laboratories, Inc"},{"orgOrder":0,"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"iECURE Receives FDA Orphan Drug Designation for GTP-506, an Investigational Gene Editing Product Candidate for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"iECURE"},{"orgOrder":0,"company":"Attralus","sponsor":"University of Tennessee Graduate School of Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus Announces New Preclinical Data Demonstrating Novel Human Chimeric Antigen Receptor-Macrophages (CAR-M) for Potential Amyloid Removal at 18th International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"orgOrder":0,"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus Presents In Vivo Preclinical Data for AT-02 Demonstrating Significant Amyloid Reduction at 18th International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Moderna Reviews Clinical Trial Programs Across Portfolio at 2022 R&D Day","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Vaccine","graph2":"Moderna Therapeutics"},{"orgOrder":0,"company":"AmideBio","sponsor":"NIDDK","pharmaFlowCategory":"D","amount":"$2.7 million","upfrontCash":"Undisclosed","newsHeadline":"AmideBio Awarded SBIR Phase IIB Grant to Support Pre-Clinical Development of its Long-Acting Stable Glucagon Analog for the Treatment of Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Peptide","graph2":"AmideBio"},{"orgOrder":0,"company":"Allomek","sponsor":"Camac Partners","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Pasithea Therapeutics Acquires AlloMek Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Allomek"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Selecta Biosciences Announces Three Presentations at the Upcoming 29th Annual European Society of Gene and Cell Therapy (ESGCT) Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Selecta Biosciences"},{"orgOrder":0,"company":"Chameleon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chameleon Biosciences Presents Poster on AAV Gene Therapy Redosing Success at ESGCT","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Chameleon Biosciences"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene and Cell Therapy Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Solid Biosciences Reports Additional Preclinical Data Demonstrating that its Novel Capsid, AAV-SLB101, Provides Superior Transduction Efficiency and Enhanced Distribution to Skeletal Muscle","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Solid Biosciences"},{"orgOrder":0,"company":"Tavanta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Tavanta Therapeutics to Present Preclinical Data for TAVT-135 at the North American Cystic Fibrosis Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Tavanta Therapeutics"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Janssen Pharmaceutical","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protagonist Therapeutics Announces Poster Presentations at the American Society of Hematology 2022 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Protagonist Therapeutics"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Elixirgen Therapeutics to Present New Data Showing Successful Ex Vivo Telomere Elongation with EXG-34217 in a Phase 1\/2 Clinical Trial at the 64th ASH Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Elixirgen Therapeutics"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Announces Positive Preclinical Data for PGN-EDO53, PGN-EDO45 and PGN-EDO44, Three Novel Duchenne Muscular Dystrophy Candidates","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"PepGen"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"Tenaya Therapeutics Announces Proposed Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Tenaya Therapeutics"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"Tenaya Therapeutics Announces Closing of Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Tenaya Therapeutics"},{"orgOrder":0,"company":"Vyant Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vyant Bio Presents Key Takeaways from Platform and Poster Presentations at Neuroscience 2022 Hosted by the Society for Neuroscience","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Undisclosed","graph2":"Vyant Bio"}]

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Tenaya Therapeutics

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Tenaya Therapeutics Announces Closing of Public Offering

Details:

TN-201 is a potentially curative gene therapy intended to address genetic hypertrophic cardiomyopathy (gHCM) caused by mutations in the Myosin Binding Protein C3 (MYBPC3) gene, that is currently in IND-enabling studies.

Lead Product(s): TN-201

Therapeutic Area: Genetic Disease Product Name: TN-201

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Morgan Stanley

Deal Size: $75.0 million Upfront Cash: Undisclosed

Deal Type: Public Offering November 21, 2022

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Tenaya Therapeutics

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Tenaya Therapeutics Announces Proposed Public Offering

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TN-201 is a potentially curative gene therapy intended to address genetic hypertrophic cardiomyopathy (gHCM) caused by mutations in the Myosin Binding Protein C3 (MYBPC3) gene, that is currently in IND-enabling studies.

Lead Product(s): TN-201

Therapeutic Area: Genetic Disease Product Name: TN-201

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Morgan Stanley

Deal Size: $75.0 million Upfront Cash: Undisclosed

Deal Type: Public Offering November 16, 2022

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Vyant Bio

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Vyant Bio Presents Key Takeaways from Platform and Poster Presentations at Neuroscience 2022 Hosted by the Society for Neuroscience

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Rett syndrome (RTT) is a progressive neurodevelopmental disorder caused by mutations in the X-linked gene MECP2. One of the challenges developing therapeutics for RTT has been the lack of a screening system that recapitulates the underlying human disease pathophysiology.

Lead Product(s): Undisclosed

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Preclinical Product Type: Undisclosed

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 16, 2022

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PepGen

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PepGen Announces Positive Preclinical Data for PGN-EDO53, PGN-EDO45 and PGN-EDO44, Three Novel Duchenne Muscular Dystrophy Candidates

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In non-human primates (NHPs), high levels of exon 53 skipping were observed after a single dose of PGN-EDO53 – almost 7-times higher than those observed for a comparator peptidephosphorodiamidate morpholino oligonucleotide (PPMO) conjugate molecule used as a positive control

Lead Product(s): PGN-EDO53

Therapeutic Area: Genetic Disease Product Name: PGN-EDO53

Highest Development Status: Preclinical Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 14, 2022

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Elixirgen Therapeutics

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Elixirgen Therapeutics to Present New Data Showing Successful Ex Vivo Telomere Elongation with EXG-34217 in a Phase 1/2 Clinical Trial at the 64th ASH Annual Meeting

Details:

EXG-34217 is an autologous cell therapy for telomere biology disorders with bone marrow failure that uses Elixirgen Therapeutics’ proprietary ZSCAN4 technology to extend the telomeres of the patients.

Lead Product(s): EXG-34217,EXG-001

Therapeutic Area: Genetic Disease Product Name: EXG-34217

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 07, 2022

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Protagonist Therapeutics

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Protagonist Therapeutics Announces Poster Presentations at the American Society of Hematology 2022 Annual Meeting

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Rusfertide Analog-PN23114 as a Hepcidin Mimetic Provides Efficacy Benefits in Conjunction with Phlebotomy in Mouse Model for Hereditary Hemochromatosis.

Lead Product(s): Rusfertide Analog

Therapeutic Area: Genetic Disease Product Name: PN23114

Highest Development Status: Preclinical Product Type: Peptide

Partner/Sponsor/Collaborator: Janssen Pharmaceutical

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 03, 2022

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Tavanta Therapeutics

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Tavanta Therapeutics to Present Preclinical Data for TAVT-135 at the North American Cystic Fibrosis Conference

Details:

TAVT-135, a cell-penetrating peptide conjugate, is a novel pan-genotypic chloride ion transporter intended to restore chloride ion balance and mucus hydration in patients with cystic fibrosis (CF).

Lead Product(s): TAVT-135

Therapeutic Area: Genetic Disease Product Name: TAVT-135

Highest Development Status: Preclinical Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 28, 2022

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Solid Biosciences

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Solid Biosciences Reports Additional Preclinical Data Demonstrating that its Novel Capsid, AAV-SLB101, Provides Superior Transduction Efficiency and Enhanced Distribution t...

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SGT-003 is Solid's next-generation AAV gene transfer therapy candidate that utilizes a rationally designed, novel muscle-tropic AAV capsid, called AAV-SLB101, to deliver Solid’s proprietary and differentiated nNOS microdystrophin for the treatment of Duchenne.

Lead Product(s): AAV-SLB101

Therapeutic Area: Genetic Disease Product Name: SGT-003

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 17, 2022

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Allomek

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Pasithea Therapeutics Acquires AlloMek Therapeutics

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CIP-137401 is a small molecule allosteric inhibitor of MEK 1/2, a key kinase in the Ras-Raf-MEK-ERK signaling pathway. Existing MEK inhibitors are marketed for a range of diseases, providing evidence for the value of regulating MEK as a drug target.

Lead Product(s): CIP-137401

Therapeutic Area: Genetic Disease Product Name: CIP-137401

Highest Development Status: Preclinical Product Type: Small molecule

Partner/Sponsor/Collaborator: Camac Partners

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Acquisition October 13, 2022

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Editas Medicine

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Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene and Cell Therapy Annu...

Details:

EDIT-103 is a CRISPR/Cas9-based experimental medicine in preclinical development for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive form of retinal degeneration.

Lead Product(s): EDIT-103

Therapeutic Area: Genetic Disease Product Name: EDIT-103

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 13, 2022

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