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Lead Product(s): SRP-9003
Therapeutic Area: Musculoskeletal Product Name: SRP-9003
Highest Development Status: Phase I/ 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 March 18, 2021
Details:
SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins.
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Lead Product(s): HST-003
Therapeutic Area: Musculoskeletal Product Name: HST-003
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: U.S. Department of Defense
Deal Size: $2.0 million Upfront Cash: Undisclosed
Deal Type: Funding September 16, 2020
Details:
The funding supports Phase 1/2 clinical trial of HST-003 for regeneration of cartilage in the knee led by Histogen. The trial is designed to evaluate HST-003 in combination with a microfracture procedure to treat recent focal cartilage defects in the knee caused by injury.
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Lead Product(s): Dynamin 101
Therapeutic Area: Musculoskeletal Product Name: DYN101
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 30, 2020
Details:
U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2), for the treatment of Myotubular and Centronuclear Myopathies (CNM).
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Lead Product(s): DYN101
Therapeutic Area: Musculoskeletal Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 05, 2020
Details:
This clinical trial, based on Ionis' technology platform, is the first investigational study testing an antisense medicine to treat Centronuclear Myopathies.
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