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Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead Pharmaceuticals to Participate in Upcoming November 2021 Conferences","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Arrowhead Pharmaceuticals"},{"company":"Sarepta Therapeutics","sponsor":"Lysogene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lysogene Announces Termination of License Agreement With Sarepta for LYS-SAF302 Program, Regaining Global Commercial Rights","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Sarepta Therapeutics"},{"company":"Agtc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AGTC Exceeds Enrollment Target in SKYLINE Trial of AGTC-501 for the Treatment of X-linked Retinitis Pigmentosa (XLRP)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Agtc"},{"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Anavex Life Sciences Provides Supplemental Information on ANAVEX\u00ae2-73 AVATAR Phase 3 Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Anavex Life Sciences"},{"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Olipudase alfa Shown to Provide Sustained Improvement Across Multiple Clinical Manifestations of ASMD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Provides Update for Planned NDA Resubmission for Arimoclomol for the Treatment of Niemann-Pick disease type C in the United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"company":"CytRx Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CytRx Highlights Orphazyme's Update on Planned NDA Resubmission for Arimoclomol in Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"CytRx Corporation"},{"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme to Present Additional Data on Arimoclomol\u2019s Mechanism of Action During 2022 Annual WORLDSymposium\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Provides Update on Efficacy, Safety and Timelines of the AAVance phase 2\/3 Clinical Trial With Gene Therapy LYS-SAF302 For the Treatment of MPSIIIA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ 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Application for Arimoclomol for the Treatment of Niemann-pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"company":"Agtc","sponsor":"Cantor Fitzgerald & Co.","pharmaFlowCategory":"D","amount":"$9.8 million","upfrontCash":"Undisclosed","newsHeadline":"AGTC Announces Proposed Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Agtc"},{"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces First Patient Dosed in Pivotal Phase 2\/3 Clinical Study of Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"company":"Agtc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Positive 18-Month Data from Ongoing Phase 1\/2 Clinical Study of AGTC-501 Presented at the Association for Research in Vision and Ophthalmology 2022 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene 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II\/ Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharvaris"},{"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GBT Initiates Phase 2\/3 Clinical Trial of GBT601 in Patients with Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Global Blood Therapeutics"},{"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals to Present Data from Phase 1 REN001 Limb Immobilization Study at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Reneo Pharmaceuticals"},{"company":"Agtc","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AGTC Announces Pricing of $10 Million Underwritten Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Agtc"},{"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"China CDE Grants Breakthrough Therapy Designation (BTD) to Neurophth's NR082 in LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"},{"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Reports Positive Results from REN001 Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Study and Provides Additional Development Updates","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Reneo Pharmaceuticals"},{"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Announces Poster Presentations at the International Network for Fatty Acid Oxidation Research and Management Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Reneo Pharmaceuticals"},{"company":"Denali Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Denali Therapeutics Announces New Interim Data from Phase 1\/2 Study of DNL310 (ETV:IDS) in MPS II (Hunter Syndrome) at SSIEM 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Denali Therapeutics"},{"company":"Forma Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","amount":"$1,100.0 million","upfrontCash":"$1,100.0 million","newsHeadline":"Novo Nordisk to Acquire Forma Therapeutics and Expand Presence in Sickle Cell Disease and Rare Blood Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Establishes Patient Specific Website for Ongoing Netherton Syndrome Clinical Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"}]
Details:
Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.
Lead Product(s):
Olipudase Alfa
Therapeutic Area: Genetic Disease
Product Name: GZ402665
Highest Development Status: Phase II/ Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
February 09, 2022
Details:
The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.
Lead Product(s):
Venglustat
Therapeutic Area: Genetic Disease
Product Name: GZ402671
Highest Development Status: Phase II/ Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 01, 2021
Details:
Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.
Lead Product(s):
Olipudase alfa
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase II/ Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
January 30, 2020
Details:
QRX003 is Quoin’s lead product candidate and has the potential to become the first approved treatment for Netherton Syndrome. Quoin intends to self-commercialize QRX003, and its other product candidates, in the US and Europe.
Lead Product(s):
QRX003
Therapeutic Area: Genetic Disease
Product Name: QRX003
Highest Development Status: Phase II/ Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 13, 2022
Details:
Setrusumab (UX143) is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway that inhibits the activity of bone-forming cells.
Lead Product(s):
Setrusumab
Therapeutic Area: Genetic Disease
Product Name: UX143
Highest Development Status: Phase II/ Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Mereo BioPharma Group
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 09, 2022
Details:
The acquisition of Forma Therapeutics, including its lead development candidate, etavopivat (FT-4202), is aligned with Novo Nordisk’s strategy to complement and accelerate its scientific presence and pipeline in haemoglobinopathies.
Lead Product(s):
Etavopivat
Therapeutic Area: Genetic Disease
Product Name: FT-4202
Highest Development Status: Phase II/ Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Novo Nordisk
Deal Size: $1,100.0 million
Upfront Cash: $1,100.0 million
Deal Type: Acquisition
September 01, 2022
Details:
The safety profile of DNL310 (Recombinant Iduronate 2-sulfatase) with up to 85 weeks of dosing remains similar to standard of care. The most frequent treatment-emergent adverse events (TEAEs) were infusion related reactions (IRRs).
Lead Product(s):
Recombinant Iduronate 2 Sulfatase,Idursulfase
Therapeutic Area: Genetic Disease
Product Name: DNL310
Highest Development Status: Phase II/ Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 31, 2022
Details:
REN001 is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in clinical development for two rare genetic mitochondrial diseases : primary mitochondrial myopathies (PMM) and long-chain fatty acid oxidation disorders (LC-FAOD).
Lead Product(s):
REN001
Therapeutic Area: Genetic Disease
Product Name: REN001
Highest Development Status: Phase II/ Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 29, 2022
Details:
REN001 is a potent and selective PPARδ agonist currently in clinical development for two rare genetic mitochondrial diseases that typically present with myopathy and have high unmet medical needs: primary mitochondrial myopathies and long-chain fatty acid oxidation disorders.
Lead Product(s):
REN001
Therapeutic Area: Genetic Disease
Product Name: REN001
Highest Development Status: Phase II/ Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
July 26, 2022
Details:
NR082 (rAAV2-ND4), a recombinant adeno-associated viral vector, serotype 2, containing human ND4 codon-optimized gene under the control of the cytomegalovirus promoter and enhancer, is a novel gene therapy.
Lead Product(s):
rAAV2-ND4
Therapeutic Area: Genetic Disease
Product Name: NR082
Highest Development Status: Phase II/ Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
July 15, 2022
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