


Details:
IMM01-STEM, a secretome product derived from partially differentiated pluripotent stem cells that contains regenerative molecules, in participants with muscle atrophy related to knee osteoarthritis (KOA).
Lead Product(s): IMM01-STEM
Therapeutic Area: Musculoskeletal Product Name: IMM01-STEM
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 January 24, 2023
Details:
AOC 1020 aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in patients with FSHD. AOC 1020 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA.
Lead Product(s): AOC 1020
Therapeutic Area: Musculoskeletal Product Name: AOC 1020
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 January 18, 2023
Details:
The scope of work supported by this grant award includes nonclinical toxicity testing of SX600 and manufacturing process development and scale-up activities. Together, these activities will position SX600 for future Phase 3 clinical trials.
Lead Product(s): Dexamethasone
Therapeutic Area: Musculoskeletal Product Name: SX600
Highest Development Status: Phase I/ Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Department of Defense
Deal Size: $5.0 million Upfront Cash: Undisclosed
Deal Type: Funding April 26, 2022
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.
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:
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).
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:
This clinical trial, based on Ionis' technology platform, is the first investigational study testing an antisense medicine to treat Centronuclear Myopathies.
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