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- NeuroOne received ISO 13485 certification, enabling international commercialization of its OneRF Brain and Trigeminal Neuralgia Ablation Systems and Evo diagnostic lines. - Third-quarter fiscal 2026 product revenue reached $2.0 million, up 16% year over year, with product orders of $2.7 million, up 43%. - The Journal of Neurosurgery published a Mayo Clinic Jacksonville study showing OneRF procedures may be performed without interfering with implanted neuromodulation hardware. - NeuroOne's robotic-assisted StereoCED intracranial drug delivery platform is expected to be ready for investigational human clinical studies by the end of September.
- uniQure was the standout contributor to abrdn Life Sciences Investors in Q2 2026, with shares re-rating dramatically following a key FDA regulatory decision. - The FDA agreed that the three-year AMT-130 data set in Huntington's disease could serve as the primary basis for a biologics license application under accelerated approval. - The fund's equity portion rose (gross of fees) and outperformed its benchmark, the NASDAQ Biotechnology Index, during the quarter. - The invested balance grew over the quarter, with new capital directed toward earlier-stage science and the new-issue window.
- Biotech industry leaders are calling for the FDA to ensure drug reviews remain grounded in scientific expertise rather than political influence following significant leadership turnover. - The FDA has lost more than 3,000 employees under the Trump administration, with the main drug-review arm cycling through six different leaders since January 2025. - Former FDA Commissioner Scott Gottlieb urged reinstating the standard that medical product centers be led by experienced scientific and clinical experts in a JAMA editorial. - The agency has announced plans to fill 2,200 open positions and accelerate clinical trial timelines, with a particular focus on maintaining U.S. leadership in cell therapy.
- BBM-P002, a dual-target AAV gene therapy delivering TH and AADC directly to the striatum, demonstrated a clean safety profile and improved motor function in a 10-patient Phase 1 trial published in Nature Medicine. - The therapy aims to create a self-contained dopamine factory in the brain, bypassing the need for surviving dopaminergic neurons in patients with moderate-to-advanced Parkinson's disease. - FDA regulatory precedent from Kebilidi's 2023 approval suggests that brain-delivered AAV therapies require years of follow-up data, and the agency now expects durability endpoints and long-term monitoring plans before Phase 2 begins. - The dual-target architecture introduces dose-response attribution challenges that sponsors must address early, as the FDA will question which gene product drives clinical observations without comparator cohorts isolating each gene's contribution.
- Acting FDA Commissioner Kyle Diamantas met with rare disease advocacy organizations on June 3 to rebuild trust following former Commissioner Marty Makary's tenure, which saw several setbacks for rare disease drugmakers. - Jeff Allen, CEO of Friends of Cancer Research, described the meeting as a "breath of fresh air" and praised Diamantas for exhibiting "real leadership" by listening to the rare disease community. - Advocacy groups pressed for greater regulatory clarity, novel clinical trial approaches, and consistency from the FDA for treatments targeting small patient populations. - The meeting comes as the White House reviews candidates for permanent FDA commissioner, with former commissioner Norman "Ned" Sharpless reportedly among those under consideration.
- HDBuzz published 100 articles in 2025, doubling 2024 output and more than tripling its historical average, reflecting accelerating HD research and clinical trial activity. - The nonprofit reached 329,000 unique visitors and generated over 630,000 page views while maintaining zero pharmaceutical funding and no paywalls. - Six HDBuzz Prize winners and five HD-CAG fellows were mentored, investing in the next generation of Huntington's disease science communicators. - HDBuzz became an independent 501(c)(3) nonprofit on January 1, 2026, and raised $23,024 from 276 donors during its spring Fund the Buzz campaign.
- Kurma Partners closed its Biofund IV at €215 million, a substantial specialist fund focused on early-stage European biotech company creation despite falling short of its original €250 million target. - The fund plans 16 to 20 investments with 80% of capital directed toward companies developing new therapeutic solutions for diseases with high unmet need. - Biofund IV has already deployed capital into multiple companies including Memo Therapeutics, Avidicure, EvlaBio, Nuevocor, Elkedonia, and Laigo Bio across diverse therapeutic areas. - Kurma's track record includes three major Biofund III exits: Amolyt Pharma acquired by AstraZeneca for up to $1.05 billion, Corlieve Therapeutics acquired by uniQure, and Emergence Therapeutics acquired by Eli Lilly.
- Five-year data from the Phase 3 HOPE-B trial published in the New England Journal of Medicine confirm HEMGENIX's durable efficacy, with 94% of patients remaining free from continuous prophylaxis treatment. - Mean factor IX activity levels remained strong at 36.1% at year five, while bleeding rates were reduced by approximately 90% compared to baseline prophylaxis treatment. - The gene therapy demonstrated a favorable safety profile with no serious treatment-related adverse events, reinforcing its potential as a transformative one-time treatment for adults with hemophilia B.
- Multiple biotechnology companies including UniQure, Biohaven, and Capricor have faced unexpected FDA reversals on previously agreed-upon evidence requirements for drug approvals since July 2024. - UniQure's Huntington's disease gene therapy AMT-130, which showed 75% disease slowing in pivotal trials, had its biologics license application timeline thrown into uncertainty after FDA changed its stance on acceptable evidence. - The regulatory instability coincides with significant leadership changes at FDA, with the Center for Drug Evaluation and Research changing hands four times since early 2025 and philosophical differences emerging between current and previous FDA leadership. - Industry analysts suggest these reversals may signal a shift toward stricter regulatory standards, requiring more compelling risk-benefit profiles despite companies having aligned with FDA on trial designs and statistical analyses.
- The FDA notified uniQure that data from Phase I/II studies of AMT-130, an investigational gene therapy for Huntington's disease, did not provide sufficient evidence to support a Biologics License Application submission. - uniQure disclosed that the FDA no longer agrees that the existing Phase I/II study data may be adequate to provide primary evidence for BLA approval, making the timing of the submission unclear. - Following the November 3, 2025 announcement, uniQure's stock price plummeted over 50%, falling from $67.69 to $34.29 in a single trading session. - The regulatory setback represents a significant blow to the development of AMT-130 as a potential treatment for Huntington's disease patients.
