Dallas, Texas – Taysha Gene Therapies touted positive early-stage data last month for its gene therapy to treat Rett syndrome, which has become the company’s main focus after cutting other programs and laying off 35% of its staff. If successful, the candidate for Rett syndrome has the capability to put...
Latest News
Washington, DC – Advances in antibody-drug conjugates, including novel payloads and new targets, will be featured at the American Association for Cancer Research’s annual conference, taking place this week in San Diego, where companies, including Elevation Oncology and Tubulis, will present new data from their respective programs. The first antibody-drug...
Washingon, DC – After a years-long drought, progress in the amyotrophic lateral sclerosis (ALS) space began to accelerate with the 2022 approval of Amylyx’s Relyvrio and Biogen and Ionis’ Qalsody in 2023. But now, that progress appears to have stalled. Earlier this month, Amylyx announced it would pull Relyvrio from...
Washington, DC – Over the last few years, we’ve witnessed a significant shift in the regulatory space for rare disease therapeutics, with the FDA and other authorities making conscious efforts to implement much-needed regulatory flexibility to accommodate the practical challenges of trial design for these conditions. For small biotechs and...
Washington, DC – Despite having not yet received full approval, Travere Therapeutics’ first-in-class endothelin and angiotensin II receptor antagonist Filspari—which received the FDA’s accelerated approval in February 2023—is already making strides in treating IgA nephropathy. A rare autoimmune disease, IgA nephropathy occurs when immunoglobulin A accumulates in the kidneys, leading...
New York, NY – Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials. The last decade has fundamentally changed how clinicians treat spinal muscular atrophy. In 2016, Biogen’s Spinraza became the FDA-approved treatment for SMA,...
Washington, DC – The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published Wednesday in the Journal of the American...
SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say. Every drug approval is a monumental occasion worthy of celebration. But for SpringWorks Therapeutics, the approval...
San Diego, Calif. – The American Association for Cancer Research’s annual conference kicks off Friday as more than 20,000 attendees descend on San Diego. Analysts and researchers who spoke with BioSpace said they’re looking forward to early scientific data, and antibody-drug conjugates (ADCs) will be a key focus. Stay tuned to...
Basel, Switzerland – The past two years have seen a pair of new treatments approved for a particularly intractable neurodegenerative disease—amyotrophic lateral sclerosis. But while Amylyx’s Relyvrio and Biogen’s Qalsody have crossed the FDA finish line, others have stumbled in a regulatory space that experts say is still evolving. The...