Neurodegenerative disease
UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp.
The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took issue with were filed in mid-2024.
Korsana’s lead program uses a next-generation shuttling technology to improve delivery into the brain and lower the incidence of amyloid-related imaging abnormalities.
Analysts, investors and scientists are eager for Biogen’s 2026 BIIB080 readout. Even if successful, executives warn that there are many more steps before the Alzheimer’s therapy could reach the market.
The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing manufacturing issues. Ultragenyx Pharmaceutical resubmitted its application ten days ago, expecting a six month review time.
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
After years of contraction, investors see biotech reentering a growth cycle driven by scientific progress, asset quality and renewed conviction in oncology, obesity and neuroscience innovation.
Roche’s obesity candidate achieves 22.5% weight loss in Phase II; Moderna pulls the plug on late-stage vaccine trials as Health Secretary Robert F. Kennedy’s anti-vaccine policies and rhetoric continues; and embattled gene therapy maker Sarepta announces new data in Duchenne muscular dystrophy.
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
Fueled by advances in biomarkers, brain mapping, and AI, investment in neuro and CNS innovation shows no signs of slowing, even amid costly setbacks. BioSpace spoke to investors at JPM26 to get their take on recent bets in new therapies and neurotech.
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