With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial at the Muscular Dystrophy Association Clinical and Scientific Conference (MDA) in Orlando, Fla., where Bridgebio Pharma Inc., Capricor Therapeutics Inc., and Solid Biosciences Inc. also had clinical findings to talk about.

Qyuns Therapeutics Co. Ltd. has moved closer to its first commercial product after China’s National Medical Products Administration (NMPA) accepted its NDA for IL-17 antibody crusekitug (QX-002N) for treating ankylosing spondylitis (AS), a chronic inflammatory disease that affects the spine and sacroiliac joints.

The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.

Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease.

Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease.

Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the Freedom2-DM1 phase II trial, a multiple ascending-dose, randomized, placebo-controlled experiment in myotonic dystrophy type 1 (DM1).

Innovacell Inc. launched a ¥14.16 billion (US$91.2 million) stock sale on the Tokyo Stock Exchange Feb. 24, ending a near two-year lull of biotech listings in Japan while signaling a dynamic year ahead for cell-based therapeutics.

Med-tech companies with an AI component in their solutions will certainly find investors willing to back them. AI after all, is being used to develop more effective, smarter technologies. However, investors will only deploy capital into innovations that address genuine clinical needs. The aging population is driving interest in devices targeting cardiovascular and musculoskeletal disorders, and other solutions geared toward neurological conditions, women’s health and diagnostics are also attracting investor attention.

In the inflamed joints of rheumatoid arthritis, CD4+ T lymphocytes accumulate lipid droplets that make them vulnerable and promote their death, thereby amplifying joint inflammation. A study led by scientists at Mayo Clinic and Stanford University suggests that blocking the formation of these lipid droplets or their contents could offer a therapeutic strategy for this condition.

The variety of organoids that can be developed in vitro is enabling major advances. Depending on the type of tissues and the research goals, these small 3D cell-based structures that mimic real tissue offer certain advantages over animal models. Scientists at the University of Padova in Italy have created human neuromuscular organoids to reproduce cancer-induced muscle cachexia, a condition that murine models do not accurately replicate.