Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
uniQure will seek approval for Huntington’s disease gene therapy after switchup at FDA
uniQure announced that they will move forward with submitting a Biologics License Application (BLA) to the FDA for their gene therapy to treat Huntington’s disease. The news follows the FDA’s prior rejection of their data package back in March, which a former top FDA official called “truly evil.” The decision was part of a string of rejections under former FDA Director of the Center for Biologics Evaluation and Research, Vinay Prasad. After Prasad left the FDA for a second time in April and FDA Commissioner Martin Makary’s exit in May, FDA officials met with uniQure and said that they will now accept a three-year analysis from their Phase 1/2 trial as the primary basis for their BLA. That trial showed that their one-time gene therapy led to a 75 percent slowing of disease at 36 months at the higher dose. UniQure’s stock rose 70 percent after the announcement in anticipation of the possible upcoming FDA approval. “The consistency and strength of the clinical data generated to date give us great confidence in the product's potential to make a meaningful difference for patients,” said Matt Kapusta, CEO at uniQure. – Allison Whitten
Non-viral gene therapy shows early promise in Duchenne muscular dystrophy
Spot Biosystems has emerged from stealth with $40 million in venture financing and early clinical evidence supporting what it says is the first non-viral delivery of full-length dystrophin to human skeletal muscle, marking a potential breakthrough for patients with Duchenne muscular dystrophy (DMD). In a study published in Nature Biomedical Engineering, the company reported that engineered extracellular vesicles safely and repeatedly delivered full-length dystrophin in mice and non-human primates, improving muscle strength without signs of liver, kidney, or cardiac toxicity. Early results from an ongoing first-in-human investigator-initiated trial in pediatric DMD patients showed dystrophin increases of more than 1,000 and 2,000 percent after one month of dosing, alongside sustained functional improvements, representing the first reported case of non-viral, full-length dystrophin delivery in humans. The findings offer a potential alternative to viral gene therapies, which are limited by cargo size and immune-related safety concerns, and provide early clinical validation for Spot Biosystems’ extracellular vesicle–based platform as it advances its DMD program toward broader clinical development. – Bree Foster
Biogen bolsters immunology pipeline with up to $1B RayThera acquisition
Biogen is strengthening its immunology pipeline through a deal to acquire private biotech RayThera for up to $1 billion, including upfront and milestone-based payments. The acquisition adds a portfolio of small molecule anti-inflammatory candidates targeting immune-mediated diseases, led by a program expected to enter Phase 1 testing in early Q3 2026. The move reflects Biogen's continued effort to diversify beyond its core neuroscience business and expand its presence in immunology through external innovation. Pending regulatory approvals, the transaction is expected to close in the third quarter of 2026, after which Biogen will lead development, manufacturing, and commercialization of the acquired assets. – Andrea Corona
CRISPR treatment shows durable control of hereditary angioedema
Intellia Therapeutics reported positive Phase 3 data showing that a single dose of its in vivo CRISPR therapy lonvoguran ziclumeran (lonvo-z) sharply reduced attacks in patients with hereditary angioedema, a rare genetic disorder marked by recurrent, unpredictable, and sometimes life-threatening swelling that typically requires lifelong preventive treatment. Results from the global HAELO trial, published in The New England Journal of Medicine, showed an 87 percent reduction in mean monthly attacks compared to placebo, with 62 percent of treated patients remaining attack-free and off prophylactic therapy for six months. Lonvo-z uses in vivo CRISPR-Cas9 gene editing to permanently inactivate the KLKB1 gene in the liver, lowering kallikrein levels after a single infusion and offering the potential for durable disease control without chronic dosing. Safety was favourable, with mostly mild to moderate infusion-related reactions and no serious adverse events reported. Intellia Therapeutics has begun a rolling Biologics License Application with the FDA and expects a US launch in the first half of 2027. – Bree Foster
FDA approves Colorado’s plan to import drugs from Canada
In a cost-saving measure, Colorado became the second state after Florida to receive approval to import medications from Canada. Florida has not yet begun their program after getting approval in 2024, and the press release states that “Colorado still faces the critical issue of procuring a supply of eligible drugs from manufacturers and urges our federal partners to take a productive role in facilitating manufacturer cooperation with the Program.” Canada also previously announced that they will not send over medications if it would lead to a shortage back home. Colorado’s news release also states that the program could save Coloradans $46 million over three years if successful. The state released a list of approved drugs to begin importing that includes Novo Nordisk’s Ozempic, Vertex Pharmaceuticals’ Trikafta, and Gilead Sciences’ Biktarvy. – Allison Whitten
