Ascidian Therapeutics Inc. secured $50 million in series A funding from its founder Apple Tree Partners to take a therapy based on its RNA exon editing technology into a first-in-human trial in ABCA4 retinopathy and to advance additional programs in neuromuscular, neurological, and rare disease indications.
Cabaletta Bio Inc. has announced CABA-201, a newly designed, fully human CD19 chimeric antigen receptor (CAR) containing a 4-1BB co-stimulatory domain.
Endpoint Health Inc. has announced a new precision immunology program targeting inflammation mediated by tissue factor (TF)-dependent intracellular signaling.
The wholesale acquisition cost for Amylyx Pharmaceuticals Inc.'s new U.S. FDA-approved amyotrophic lateral sclerosis (ALS) treatment, Relyvrio, has been set at about $158,000 for the first year’s treatment. It jumps to about $163,000 in the second year, the company said, a change that would move it closer to the cost of competitor Mitsubishi Tanabe Pharma Corp.'s Radicava (edaravone), which costs about $165,000 annually. A 28-day prescription will cost $12,504.
Solid Biosciences Inc. found a way to continue its Duchenne muscular dystrophy push, inking a deal to acquire privately held Aavantibio Inc., at the same time nailing down $75 million by way of a private placement with institutional investors.
Eli Lilly & Co. has identified new fibroblast growth factor receptor 3 (FGFR3) inhibitors reported to be useful for the treatment of achondroplasia, cancer, pulmonary fibrosis, systemic scleroderma and thanatophoric dysplasia, among other disorders.
A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.
A simple injection of muscle tissue could control glucose in patients with type 2 diabetes (T2D). Genetic modification of skeletal muscle and subsequent intramuscular implantation could increase blood sugar absorption and become an effective and long-lasting treatment for this pathology. “We took mice satellite cells and we genetically altered to overexpress GLUT4,” Hagit Shoyhet, researcher at the Levenberg lab of stem-cell and tissue engineering, Technion (Israel), said at the European Association for the study of Diabetes (EASD) 58th Annual Meeting.
It failed to meet the primary endpoint at six months, but the European chief investigator for Biogen Inc.’s phase III trial of tofersen in treating amyotrophic lateral sclerosis (ALS) now describes the study as “trailblazing,” following a six-month open label extension.
Virios Therapeutics Inc. said most likely COVID-19 had a hand in the phase IIb failure of IMC-1 (famciclovir + celecoxib), a dual COX-2/COX-1 inhibitor for treating fibromyalgia. The drug failed to hit statistical significance in dampening pain severity when compared to placebo (p=0.302).
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