[{"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces Initiation of Planned Pivotal Clinical Trial for Myotonic Dystrophy Following $35m Fund Raise","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma Announces Additional Positive Data from Phase 2b ASTEROID Study of Setrusumab in Adults with Osteogenesis Imperfecta and Provides Update on Regulatory Progress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Mereo BioPharma Group"},{"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Receives FDA Fast Track Designation for Niemann-Pick Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"company":"Oxford Biomedica","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sanofi Returns Rights to Gene Therapies to Oxford Biomedica","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Oxford Biomedica"},{"company":"ReNeuron","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReNeuron Announces Further Positive Data in Retinal Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"ReNeuron"},{"company":"GSK","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Licenses Gene Therapy Technology from GSK","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GSK"},{"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma Receives FDA Rare Pediatric Disease Designation for Setrusumab for the Treatment of Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Mereo BioPharma Group"},{"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"company":"Mereo BioPharma Group","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","amount":"$304.0 million","upfrontCash":"$50.0 million","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Collaboration and License Agreement for Setrusumab in Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Mereo BioPharma Group"},{"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma to Host Conference Call Highlighting Top-line Clinical Data from ASTRAEUS Phase 2 Study of Alvelestat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Mereo BioPharma Group"}]
Details:
MPH-966 (alvelestat) is an oral drug that is being researched in people with alpha-1 antitrypsin deficiency (AATD) lung disease. Lung disease is the most common impact of AATD in adults.
Lead Product(s):
Alvelestat
Therapeutic Area: Genetic Disease
Product Name: MPH966
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
May 06, 2022
Details:
Ultragenyx will lead future global development of setrusumab in both pediatric and adult patients. Mereo granted Ultragenyx an exclusive license to develop and commercialize setrusumab in the US and rest of the world, excluding Europe where Mereo retains commercial rights.
Lead Product(s):
Setrusumab
Therapeutic Area: Genetic Disease
Product Name: BPS-804
Highest Development Status: Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Ultragenyx Pharmaceutical
Deal Size: $304.0 million
Upfront Cash: $50.0 million
Deal Type: Collaboration
December 17, 2020
Details:
IB1001 demonstrated a statistically significant and clinically meaningful change in both the primary and secondary endpoints.
Lead Product(s):
N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease
Product Name: IB1001
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
October 19, 2020
Details:
IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC.
Lead Product(s):
N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease
Product Name: IB1001
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 30, 2020
Details:
In Mereo’s Phase 2b ASTEROID study, setrusumab demonstrated a dose-dependent bone building effect and a trend of reduction in fractures in addition to being safe and well tolerated in adults with OI.
Lead Product(s):
Setrusumab
Therapeutic Area: Genetic Disease
Product Name: BPS-804
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
September 24, 2020
Details:
Orchard has entered into this license agreements with GSK for use of their lentiviral stable cell line technology for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.
Lead Product(s):
OTL-103
Therapeutic Area: Genetic Disease
Product Name: OTL-103
Highest Development Status: Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Orchard Therapeutics
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Licensing Agreement
July 15, 2020
Details:
The Company reports that the latest data continue to demonstrate the efficacy of the therapy (hRPC stem cell therapy) , with a clinically meaningful benefit being observed at all time-points.
Lead Product(s):
Human retinal progenitor cell therapy
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
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
June 29, 2020
Details:
Oxford Biomedica has been informed by Sanofi that following completion of a company-wide portfolio review, it intends to return to OXB the rights to the following ophthalmology gene therapy programmes: SAR422459 for Stargardt disease & SAR421869 for Usher’s Syndrome type 1b.
Lead Product(s):
SAR422459
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Sanofi
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Licensing Agreement
June 09, 2020
Details:
IB1001 (N-acetyl-L-leucine) is currently being investigated for the symptomatic, and disease-modifying, neuroprotective treatment of NPC in a multinational clinical trial (IB1001-201).
Lead Product(s):
N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
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
March 25, 2020
Details:
Setrusumab demonstrated a dose dependent increase in both failure load and stiffness at the radius at 12 months, achieving statistical significance in the high dose cohort.
Lead Product(s):
Setrusumab
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
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
January 14, 2020
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