Daily Snap - 29. September 2025

Author

Joachim Eeckhout
September 29, 2025

 

Good morning! The U.S. Advanced Research Projects Agency for Health (ARPA-H) just rolled out two new nine-figure programs to push personalized gene editing from one-off miracles toward a scalable reality. The THRIVE program will fund platforms that can generate personalized CRISPR therapies for rare diseases, while GIVE will back hospital-based manufacturing units to produce those therapies closer to patients. The effort builds on the breakthrough earlier this year when KJ Muldoon, a baby with a rare metabolic disease, received a first-of-its-kind custom CRISPR treatment and survived. With the FDA prepping a new regulatory pathway for individualized therapies, the race is on to turn this story from an exception into the rule.

Enjoy today’s read!

—Joachim E. 

SNIPPETS

What’s happening in biotech today?

🤝Dual-ADC deal: Synaffix, a subsidiary of Lonza, has entered a licensing agreement with South Korea’s Qurient to develop a dual-payload antibody-drug conjugate (ADC) for solid tumor treatment. The collaboration will merge Synaffix’s exatecan-based ADC technology with Qurient’s CDK7 inhibitor. Under the agreement, Synaffix will provide the ADC technology while Lonza manages component manufacturing, and Qurient will lead research, development, and commercialization. Dual-payload ADCs aim to enhance therapeutic efficacy and overcome resistance by delivering two cytotoxic agents with distinct mechanisms.

 🪝Generic snag: Taiwan-based generic manufacturer Lotus Pharmaceuticals has acquired a 100% equity stake in U.S.-based Alvogen in a deal valued up to $2 billion, positioning Lotus among the top 20 specialty pharmaceutical companies globally. The acquisition, orchestrated by serial entrepreneur Robert Wessman, who chairs both companies, grants Lotus access to Alvogen’s R&D, manufacturing, and commercial infrastructure in the U.S., including a key facility in Norwich, New York. The move strengthens Lotus’ global footprint and could help mitigate the impact of impending U.S. tariffs on imported pharmaceuticals. The merger reflects a continued trend of consolidation in the generics market, following similar deals involving Zentiva and Stada.

🧠Amyloid amelioration: Eli Lilly’s Alzheimer’s drug Kisunla (donanemab) has received approval from the European Commission for treating early symptomatic Alzheimer’s disease, following a previous rejection by EMA advisors over safety concerns related to amyloid-related imaging abnormalities (ARIA). The approval is limited to adults with mild cognitive impairment or mild dementia who do not carry two copies of the ApoE4 gene. Supported by data from the TRAILBLAZER-ALZ 2 and 6 trials, Kisunla showed it could significantly slow cognitive and functional decline, with sustained benefits over three years. Despite its promise, pricing challenges may affect broader adoption, particularly in markets like the UK.

💥LDL takes an L: The FDA has expanded approval of Regeneron’s Evkeeza to treat children as young as 1 year old with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder affecting about 1,300 people in the U.S. This follows previous approvals for older age groups in 2021 and 2023, reflecting a strategic effort to broaden the drug's clinical reach. Supported by data from six young patients, the drug continues to show a ~50% reduction in LDL-C levels when added to standard therapies. Despite the small market, Evkeeza benefits from orphan drug pricing, reinforcing Regeneron’s rare disease portfolio.

💊Dopamine boost: AbbVie has submitted an NDA to the FDA for tavapadon, a selective dopamine D1/D5 receptor partial agonist, as a once-daily oral treatment for Parkinson's disease. The application is supported by positive efficacy and safety data from the phase 3 TEMPO program, including three randomized trials, TEMPO-1 and TEMPO-2 in early-stage Parkinson’s and TEMPO-3 as adjunctive therapy to levodopa in patients with motor fluctuations. These studies showed significant improvements in motor function and daily living activities. Tavapadon was well tolerated, with mild to moderate adverse events. An open-label extension study is ongoing.

TOUR OPERATOR

Upcoming events