With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
A tough spring has settled into a cruel summer for Fibrogen Inc. as the company has stumbled for the third time in two months. The newest problem is top-line results showing the phase III Zephyrus-1 study of pamrevlumab, a monoclonal antibody, missed its primary endpoint for treating idiopathic pulmonary fibrosis.
A research team led by Domagoj Cikes at the Institute of Molecular Biotechnology of the Austrian Academy of Sciences (IMBA) and Josef Penninger at the University of British Columbia has discovered the critical role of the enzyme phosphate cytidylyltransferase 2 (PCYT2) in muscle health. Their findings appeared in the March 20, 2023, online edition of Nature Metabolism.
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
Investigators at University of British Columbia have reported the precise cellular populations responsible for the inability to regenerate muscle tissues in muscular dystrophy.
In the more than two years since Casma Therapeutics Inc. raised its series A and completed its new $50 million series B, the company has advanced its agonist program for treating muscular dystrophy and identified new targets.
The CRISPR genome editing technique has been used to activate a close relative of the gene that is mutated in muscular dystrophy type 1A, preventing development of the disease in mouse-model neonates and reversing symptoms in mice with established pathology.
