UniQure is preparing to seek accelerated FDA approval for its Huntington’s disease gene therapy, a move that follows the FDA’s reversal of its prior position calling for another clinical trial. This gene therapy is the latest in a growing list of rare disease treatments to receive reconsideration from an FDA that appears to be swinging back to regulatory flexibility.
UniQure plans a first quarter 2026 FDA submission for AMT-130 as a treatment for Huntington’s disease. This gene therapy delivers microRNA designed to silence the mutated gene that produces a protein driving this disorder, which so far has no FDA-approved therapies.
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The structure of Atalanta Therapeutics’ RNA interference drugs enables them to distribute broadly and deeply into brain tissue. Lead programs for Huntington’s disease and a rare inherited form epilepsy are on track to enter the clinic this year.
Novartis is securing global rights to a PTC Therapeutics Huntington’s disease drug candidate that has encouraging Phase 2 data that will be discussed with the FDA to plan a pivotal study. Last year, Novartis discontinued its Huntington’s program after safety issues emerged in Phase 2 testing.
A uniQure gene therapy for Huntington’s disease has interim clinical data showing an 80% slowing of disease progression. An accelerated approval pathway is one of the topics the company wants to discuss with the FDA.
Novartis is paying Voyager Therapeutics $100 million up front to collaborate on gene therapies for Huntington’s disease and spinal muscular atrophy. The deal builds on a relationship the companies started in 2022.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
Beth Hoffman, Origami Therapeutics CEO, talks about how the company's platform enables the discovery and development of protein degraders and conformation correctors to match the best drug to treat neurodegenerative diseases, such as Huntington's disease.
Ipsen is shoring up its neuroscience pipeline via a deal that gives it the option to license drugs that Exicure is developing for Huntington’s disease and Angelman syndrome. The programs are based on Exicure’s proprietary technology that produces nucleic acid therapies capable of delivery deep into the brain.
The companies will use a combination of more than seven years of medical record data with five years of prospective data on 5,000 MS patients, including real-world outcomes data and MRI images. Other disease areas to be explored include hematology and rare diseases.
Voyager will gain worldwide rights to its Huntington's disease therapy candidate, transfer rights to another product candidate to Neurocrine and pay Sanofi $20 million.
Healthcare payments are no longer about transactions. It’s about controlling the revenue cycle. And increasingly, that control is not sitting with ISOs.
Neurodegenerative diseases represent a particularly interesting area for wearables to come into play as research scientists seek to better understand disease progression.
“If it’s positive it’s a death sentence,” Filmmaker Marianna Palka says. “If it’s the opposite, I live like everyone else.” Palka has a 50/50 chance of developing Huntington’s disease. She watched her father become completely debilitated, to say the least, and two of her siblings have the disease. This genetic disease has been described as […]
CeNeRx BioPharma's investigational new drug application on a compound aimed at treating peripheral neuropathy has been approved by the U.S. Food and Drug Administration. The Cary, North Carolina-based company is now preparing to move forward in human trials of the compound.
Last week, CHDI Foundation, a not-for-profit research organization, said it would "initially" pay up to 50 percent of clinical costs of a drug/device therapy being developed by Medtronic and Alnylam Pharmaceutical to treat Huntington's disease. While medical device and biopharmaceutical companies are partnering more and more, the inclusion of the foundation in this trio is what's interesting.
Medtronic Inc. (NYSE:MDT) said Wednesday it will form an alliance with a pharmaceutical firm and research foundation to develop a unique drug/device therapy to treat Huntington's disease. The medical firm, based in Fridley, Minnesota, said it will partner with Alnylam Pharmaceutical Inc. (Nasdaq:ALNY) and the CHDI Foundation to deliver a cutting-edge drug technology directly to the brain using an implantable infusion system being developed by Medtronic.