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Aug 21, 2026
New research shows that many people with disease-causing gene mutations never develop illness, challenging traditional assumptions. Studies of 'modifier genes' and genetic outliers are revealing how some individuals stay healthy, potentially leading to new treatments for genetic diseases.
Aug 11, 2026
Several drugmakers saw shares fall after earnings missed expectations. Alnylam's Amvuttra sales disappointed despite growth; Neurocrine's guidance underwhelmed; AbbVie's Imbruvica and Roche's Vabysmo fell short of consensus.
May 27, 2026
The FDA replaced the two-trial approval requirement with a single pivotal trial standard, launched an AI pilot with AstraZeneca and Amgen, and extended the camizestrant review. A key FDA official is departing. AstraZeneca posted $58.74 billion in revenue.
Jul 23, 2026
Transcripta Bio raised $24M backed by Mayo Clinic and Omnimed for AI-driven neurological and neuromuscular therapies. The funds will support IND-enabling studies and clinical preparation for ASD and FSHD programs.
Jul 11, 2026
A new gene therapy platform uses the brain's glymphatic system to deliver genes to glial cells throughout the brain. It bypasses the blood-brain barrier and could enable treatments for multiple sclerosis, Huntington's disease, and rare childhood white matter disorders.
Feb 24, 2026
Myriad Genetics, uniQure, and Legend Biotech have scheduled Q4 and full year 2025 earnings calls for Feb 23, Mar 2, and Mar 10, 2026, respectively. Details include webcast access, dial-in numbers, and company background.
Aug 05, 2026
Federal courts are weighing whether the April 2026 marijuana rescheduling order was lawful. MMJ argues the Attorney General skipped required rulemaking; DEA testimony flagged omitted diversion analysis. A stay motion is pending.
Jun 18, 2026
The FDA has reversed its earlier rejection of UniQure's Huntington's disease therapy AMT-130, accepting existing trial data for accelerated review. The company plans to submit a marketing application in Q3 2026, ending a dispute over sham surgery requirements.
Jun 17, 2026
The FDA agreed uniQure's existing trial data could support an accelerated approval filing for Huntington's therapy AMT-130, a reversal from earlier positions. Meanwhile, UK-based Harness Therapeutics selected HRN001 as its lead experimental candidate, targeting the FAN1 protein to address somatic expansion of Huntington's-causing genetic repeats.
May 05, 2026
Novartis introduced new long-term social impact and sustainability targets covering access, global health, emissions, waste and water. The plan includes a 2030 access goal and net-zero across the value chain by 2040.
Apr 21, 2026
The FDA said uniQure’s Phase I/II data for Huntington’s disease gene therapy AMT-130 are not adequate for a marketing application. The agency recommended a new randomised, double-blind, sham surgery-controlled study.
Apr 19, 2026
UniQure shares fell sharply after FDA comments appeared to raise concerns about the invasive delivery method for Huntington's disease therapy AMT-130. The company had planned a 2026 BLA filing after Phase I/II data showed a 75% slowing of disease progression at 36 months.
Apr 03, 2026
A phase 3 clinical trial has launched for pridopidine as a potential ALS treatment, while AI research identified 18 FDA-approved drugs that may extend survival. Separate studies found a promising three-drug combination for sporadic ALS using new cell models.
Mar 23, 2026
Huntington's disease provides neuroscience with a uniquely clear genetic model for studying brain disorders, featuring a single identifiable mutation and predictable disease progression. The condition serves as a testing ground for cutting-edge therapies and raises fundamental questions about brain regeneration. Its scientific clarity combined with a united patient and research community makes it an invaluable resource for advancing neurological understanding.
Mar 23, 2026
Sarepta Therapeutics received FDA feedback supporting supplemental applications for AMONDYS 45 and VYONDYS 53, while New Zealand approved a Phase 1 trial for Huntington's Disease candidate SRP-1005. The company faces significant share price declines despite these developments, with analysts showing wide disagreement on valuation targets ranging from $5 to $80.
Mar 21, 2026
uniQure disclosed the FDA will not accept Phase I/II data alone for AMT-130 approval, causing shares to drop 49%. The company held regulatory meetings and reported $622.5 million in cash while investors face an April 13 class action deadline.
Mar 07, 2026
The FDA has denied or discouraged at least eight drug applications in the past year, including treatments for rare diseases, raising concerns about regulatory consistency and the future of experimental therapies.
Mar 03, 2026
The FDA informed uniQure that Phase 1/2 study data for AMT-130 in Huntington's disease is insufficient to support a biologics license application, recommending a prospective, randomized, double-blind, sham surgery-controlled study instead.
Mar 03, 2026
uniQure reports Q4 2025 results as investors await clarity on AMT-130 Huntington's disease gene therapy following FDA's November indication that Phase I/II data unlikely to support biologics license application.
Mar 02, 2026
uniQure received FDA feedback stating current AMT-130 data unlikely to support accelerated approval for Huntington's disease, while securities fraud lawsuits challenge prior disclosures. Fresh Fabry disease trial data showed elevated enzyme activity but prompted a dosing pause for safety review.
