The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.
The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
Spastic paraplegia type 47 (SPG47) is a rare congenital and neurological disorder characterized by dysfunction of the long axons in the corticospinal tract neurons that lead to progressive limb weakness and spasticity.
U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.
Capsida Biotherapeutics Inc. presented preclinical data for a new next-generation gene supplementation therapy candidate, CAP-003, for Parkinson’s disease (PD) patients with GBA1 mutations (PD-GBA).
Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in the newest episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.”
Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan.
More than half of all hearing loss cases are hereditary. Myelin protein zero-like 2, encoded by MPZL2, is widely expressed in cochlear cells in the inner ear. Mutations in MPZL2 have been identified as the second most prevalent cause of mild to moderate hereditary hearing loss.
Purespring Therapeutics Ltd. has raised £80 million (US$104.6 million) in a series B, putting it on course to be the first to take a gene therapy for a kidney disease into the clinic. The money enables the company to move the lead program, PS-002, for the treatment of IgA nephropathy to clinical proof of concept and advance programs in other complement-mediated kidney diseases, and in an undisclosed glomerular kidney disease.
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