Though it’s been used off-label for more than three decades to treat cerebrotendinous xanthomatosis, Mirum Pharmaceuticals Inc.’s chenodiol gained an official U.S. FDA nod Feb. 21 as the first drug approved specifically for treating the rare autosomal recessive lipid storage disease. Read More
Gene therapy specialist Meiragtx Holdings plc is heading for its first marketing approval following the successful treatment of 11 children with Leber amaurosis, a severe form of congenital retinal dystrophy that rendered them blind at birth. The 11 children, aged between 1 and 4 years old, all gained visual acuity following a single delivery of a correct version of the AIPL1 (aryl-hydrocarbon interacting protein-like1) gene, which codes for a photoreceptor protein in the cones and rods. Read More
As the flurry of executive orders continues to flow from the White House, leaving in its wake chaos and confusion across the overall health care sector, Tim Opler, managing director of the global health care group at investment firm Stifel, is taking the long view and urging the biopharma sector to continue focusing on innovation. Read More
Once high-flying Bluebird Bio Inc. has found a way out of its financial squeeze, as funds managed by global investment firms Carlyle and SK Capital Partners LP, along with a team of biotech executives, will be taking over the company. Read More
Several Asia biotechs this week – including Innocare Pharma Ltd., Akeso Pharmaceuticals Inc., Sanbio Co. Ltd. and Ascletis Pharma Inc. – unveiled the start of new late-stage clinical trials or interim findings from early stage studies. Read More
The U.S. FDA approved 12 drugs in January, falling below the 2024 monthly average of 19. Only three new molecular entities received approval, trailing the yearly average of just over four per month. Read More
The lung and thrombosis may play a key role in cancer and metastasis progression, according to a collaborative study led by Cornell University scientists. Read More
The Clarivate Medtech Trends to Watch in 2024 report noted the resilience of the medtech sector in the face of changing macroeconomic factors and the preparation required to navigate the market’s many complexities. It is likely that 2025 will be no different. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aptose, Assembly, Medivir, Novadip, Sellas, Thetis. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Astellas, Astrazeneca, Boost Neurosciences, Fibrogen, Gate Neurosciences, Hangzhou Polymed, Harbour, Iconovir, Insilico Medicine, Max, Metaba, Myris, Photys, Urogen. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Apellis, Arctic Vision, Biorestorative, Clearside, CSL, Genmab, Irlab, Polaritybio, Signet, Sobi. Read More
