Whole genome sequencing has substantially accelerated the pace of discovery of genes that cause rare diseases, but while this has brought the diagnostic odyssey of some patients to a conclusion, 50% to 80% remain undiagnosed after initial analysis.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Formycon, Grin, Hyloris, Kashiv, Merck & Co., Mirum, Precigen, Sanofi.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Elixirgen, Eupraxia, Medicenna, Regeneron, Terns, Yoltech.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Carsgen, Eli Lilly, Hyloris, Infex, Kiniksa, Lava, Nimbus, Oncopeptides, Organovo, Redhill, Ucarsgen, Venus.
The Trump administration dashed hopes that it would temper the Medicare price negotiations mandated by the Inflation Reduction Act when it filed the government’s brief in response to Novartis AG’s appeal to the U.S. Court of Appeals for the Third Circuit.
Qyuns Therapeutics Co. Ltd.’s monoclonal antibody targeting IL-17A, QX-002N, met both primary and secondary endpoints in a phase III trial in ankylosing spondylitis.
Nearly four years after signing its first major partnership with Takeda Pharmaceutical Co. Ltd., Bridgene Biosciences Inc. returned for a second deal with Takeda – this time focused on using its chemoproteomics platform to discover novel small molecules against immunology and neurology targets.
As Wall Street looks ahead to phase III data due soon with Mineralys Therapeutics Inc.’s lorundrostat for uncontrolled or resistant hypertension, analysts are also weighing the odds of the firm in phase II with the same compound for obstructive sleep apnea.
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