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Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Announces Positive Interim Results from a Phase 2 Trial of Infigratinib in Achondroplasia Demonstrating an Increase in Annualized Height Velocity of 1.52 cm\/year in Children 5 Years of Age and Older, and Adds 5th Cohort to Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Announces Positive Interim Results from a Phase 2 Trial of Infigratinib in Achondroplasia Demonstrating an Increase in Annualized Height Velocity of 1.52 cm\/year in Children 5 Years of Age and Older, and Adds 5th Cohort to Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"company":"Avidity Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Avidity Biosciences Enrolls Patients in the MARINA\u2122 Open-Label Extension Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 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Treatment of Friedreich\u2019s Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Reata Pharmaceuticals"},{"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope Therapeutics to Present at the Retina Forum 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Nanoscope Therapeutics"},{"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope Announces Presentations at the 22nd Euretina Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Nanoscope Therapeutics"},{"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Therapeutics Announces FDA Authorization for Phase 2 Clinical Trial of EDG-5506 for the Treatment of Duchenne Muscular Dystrophy (DMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"company":"Zynerba Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zynerba Pharmaceuticals Presents Positive Data from Phase 2 INSPIRE Trial in 22q at The Society for the Study of Behavioural Phenotypes (SSBP) 24th International Research Symposium","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Zynerba Pharmaceuticals"},{"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Therapeutics Announces Positive 4-Month Interim Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker Muscular Dystrophy (BMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope Therapeutics Announces Completion of Enrollment in STARLIGHT Phase 2 Clinical Trial of MCO-010 Optogenetic Gene Therapy for Stargardt Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Nanoscope Therapeutics"},{"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Reports Topline Results from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis (CF) 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Morgan","pharmaFlowCategory":"D","amount":"$138.0 million","upfrontCash":"Undisclosed","newsHeadline":"Edgewise Therapeutics Announces Closing of Upsized Public Offering of Common Stock, Including Full Exercise of Underwriters\u2019 Option to Purchase Additional Shares","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"company":"jCyte","sponsor":"Center for Breakthrough Medicines","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Center for Breakthrough Medicines and jCyte Announce Strategic Partnership to Manufacture jCyte\u2019s Innovative Cell Therapy for Treatment of Retinal Degenerative Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"jCyte"}]
Details:
Under the terms of the agreement, CBM will provide Phase 3 clinical trial supplies and commercial drug product following Biologics License Application (BLA) approval of jCyte’s proprietary jCell platform technology working on human retinal progenitor cells.
Lead Product(s):
Human Retinal Progenitor Cells
Therapeutic Area: Genetic Disease
Product Name: jCell
Highest Development Status: Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Center for Breakthrough Medicines
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Partnership
September 20, 2022
Details:
The total proceeds from the offering will be used for research and development of EDG-5506 and other products in clinical pipeline and for other general corporate purpose.
Lead Product(s):
EDG-5506
Therapeutic Area: Genetic Disease
Product Name: EDG-5506
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
J.P. Morgan
Deal Size: $138.0 million
Upfront Cash: Undisclosed
Deal Type: Public Offering
September 16, 2022
Details:
Evidence of activity for ELX-02 was observed, as patients with higher baseline sweat chloride levels demonstrated increased responses as indicated by SCC (p=0.00013 at Day 35).
Lead Product(s):
ELX-02,Ivacaftor
Therapeutic Area: Genetic Disease
Product Name: ELX-02
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 14, 2022
Details:
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of vMCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose).
Lead Product(s):
vMCO-010
Therapeutic Area: Genetic Disease
Product Name: vMCO-010
Highest Development Status: Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 13, 2022
Details:
EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD.
Lead Product(s):
EDG-5506
Therapeutic Area: Genetic Disease
Product Name: EDG-5506
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 11, 2022
Details:
INSPIRE trial for Zygel (Cannabidiol), in 22q achieved statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments, with excellent tolerability profile.
Lead Product(s):
Cannabidiol
Therapeutic Area: Genetic Disease
Product Name: Zygel
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 08, 2022
Details:
VX15/2503 (pepinemab) antibody blockade SEMA4D in early Huntington’s Disease analysis showed trial did not meet primary endpoints, brain imaging measures showed significant reduction in atrophy of caudate nucleus and prevented loss of metabolic activity in most brain regions.
Lead Product(s):
Pepinemab
Therapeutic Area: Genetic Disease
Product Name: VX15/2503
Highest Development Status: Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 08, 2022
Details:
AOC 1001, utilizing its AOC platform, is designed to address root cause of DM1 by reducing levels of DMPK, the disease-related mRNA, also consists of a proprietary mAB that binds to transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA).
Lead Product(s):
AOC 1001
Therapeutic Area: Genetic Disease
Product Name: AOC 1001
Highest Development Status: Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 02, 2022
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