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[{"orgOrder":0,"company":"Harvard Stem Cell Institute","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Muscular Dystrophy Collaboration Aims to Correct Muscle Stem Cells\u2019 DNA","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Harvard Stem Cell Institute"},{"orgOrder":0,"company":"Altay Therapeutics","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","amount":"$3.0 million","upfrontCash":"Undisclosed","newsHeadline":"SOLVE FSHD Invests US$3 Million in Altay Therapeutics","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Small molecule","productStatus":"Undisclosed","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Altay Therapeutics"}]
Find Novel Musculoskeletal Drugs in Discovery
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Details:
The investment will support Altay Therapeutics’ unique approach to finding an inhibitor of DUX4, a key transcription factor implicated in FSHD, that could provide an effective disease-modifying treatment.
Lead Product(s):
Undisclosed
Therapeutic Area: Musculoskeletal
Product Name: Undisclosed
Highest Development Status: Discovery
Product Type: Small molecule
Partner/Sponsor/Collaborator:
SOLVE FSHD
Deal Size: $3.0 million
Upfront Cash: Undisclosed
Deal Type: Financing
September 21, 2022
Harvard Stem Cell Institute
Details:
As per collaboration agreement, the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein.
Lead Product(s):
CRISPR/Cas9 genome
Therapeutic Area: Musculoskeletal
Product Name: Undisclosed
Highest Development Status: Discovery
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Sarepta Therapeutics
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Collaboration
January 13, 2020