Myogenica Inc., a University of Minnesota startup company, has obtained IND approval from the FDA for Myopaxon, an induced pluripotent stem cell (iPSC)-derived muscle stem cell product to regenerate skeletal muscle. A planned study will evaluate intramuscular injections of Myopaxon in non-ambulatory adult patients with Duchenne muscular dystrophy.
Researchers from the University of California San Francisco (UCSF) have found that CCN3, a hormone secreted by neurons in the brain, is responsible for maintaining bone strength during lactation.
The big advantage of cell culture to model diseases is its throughput. “You can play the disease over and over again in the dish,” Clive Svendsen told the audience at the International Society of Stem Cell Research (ISSCR) Annual Meeting held in Hamburg last week. That high throughput, however, is not particularly useful if the cell lines themselves do not accurately model the disease. Cancer cell lines are used in many cell culture experiments far beyond cancer for their ability to grow. But they are “highly abnormal,” Bill Skarnes told the audience at an innovation showcase, as well as quite unstable. “I don’t think the [HEK-293] cell line is the same in your lab as it is in the lab next door,” Skarnes said.
Phase I/II data of IDCT (rebonuputemcel), a cell therapy in development for chronic and progressive lumbar degenerative disc disease and one that has both regenerative medicine advanced therapy and fast track designations in the U.S., has been published in the International Journal of Spine Surgery for showing statistically significant improvements in pain, disability and quality of life.
Caregen Co. Ltd. has described peptides reported to be useful for the treatment of cartilage injury, fracture, intervertebral disc herniation, degenerative intervertebral disc disorder, osteoarthritis, osteomalacia and muscular injury.
Hypochondroplasia (HCH) is a skeletal dysplasia similar to achondroplasia (ACH) but with milder features, that affects particularly the ossification of proximal long bones of arms and legs. Around 70% to 80% of cases of HCH are caused by N540K alterations in the FGFR3 gene. At the recent 6th Annual Achondroplasia & Skeletal Dysplasia Research Conference, researchers from Tyra Biosciences Inc. presented preclinical proof-of-concept data of TYRA-300, an oral FGFR3-selective inhibitor in a model of HCH.
Two drugs were pushed back by the EMA last week, with a recommendation that Ocaliva, currently the only second line standard of care for treating primary biliary cholangitis, be withdrawn from the market, and a refusal to grant conditional approval for masitinib in the treatment of amyotrophic lateral sclerosis.
Work at Shanghai Meiyue Biotech Development Co. Ltd. has led to the discovery of spiro-heterocycle substituted pyrimidine compounds acting as C-C chemokine receptor type 4 (CCR4) antagonists.
Monte Rosa Therapeutics Inc. has submitted an IND application to the FDA for MRT-6160, a highly selective and orally bioavailable molecular glue degrader directed against VAV1 in development for systemic and neurological autoimmune diseases.
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