biotech2.475 min read
FDA hold stalls Regenxbio's RGX-121 refiling amid spine MRI findings
1 day ago•Source: Fierce Biotech
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2.245 min•7 days ago
Oral lac-phe pill aims to mimic exercise, preserving muscle during weight loss in early trials
Enveda reports Phase 1 results for an oral drug candidate that mimics the exercise-induced lactate-phenylalanine pathway (lac-phe) to curb appetite and preserve lean muscle, with the goal of maintaining weight loss without the GI side effects seen with GLP-1 drugs.
2.265 min•14 days ago
Infinimmune lands $75M Series A to push eczema antibodies forward
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AbCellera Bets ABCL635 as Best-in-Class Non-Hormonal Hot-Flash Therapy
Fierce Biotech•15 days ago
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FDA Rejects ITM-11 Radiopharmaceutical, Stalling Rival to Novartis Therapy
The FDA rejected ITM-11, ITM Isotope Technologies Munich’s radiopharmaceutical for gastroenteropancreatic neuroendocrine tumors, due to manufacturing issues, halting a potential rival to Novartis’ therapy; early ITM data suggested ITM-11 could extend progression-free survival (23.9 months vs 14.1 months with everolimus).
ProteinGuide enables on-the-fly conditioning of protein design models with experimental data
ProteinGuide provides an inference-time conditioning framework that lets a broad class of protein sequence generators (including masked language models like ESM3, autoregressive models like ProteinMPNN, and discrete-space diffusion/flow models) be guided by auxiliary experimental data without retraining. Built on a unifying statistical approach, it enables designs targeting properties such as stability and activity, supports multi-property Pareto optimization, and, when combined with wet-lab data, can enhance in vivo base-editor activity beyond multi-round directed evolution. The work provides code and data (Zenodo, GitHub) and a ProteinGen package to facilitate design workflows.
Peptides win FDA panel ok, CRISPR tragedy raises ethics questions, insulin supply tightens biotech spotlight
An FDA panel narrowly approved allowing compounding pharmacies to produce certain unregulated peptides—a win for RFK Jr.—while Science reports a six-year-old girl's death in China after an experimental brain-directed CRISPR therapy, prompting ethics and oversight concerns; Sanofi insulin pens are in short supply, underscoring ongoing biotech and pharmaceutical tensions.
Sonothera bets ultrasound could unlock Duchenne gene therapy
A California startup called Sonothera claims ultrasound can enable gene therapies to treat Duchenne muscular dystrophy and other genetic diseases. They’ve shown striking animal data but have no clinical data yet, and several experts remain skeptical about whether the results will translate to humans.
Biotech startup charts a GLP-1-free path to obesity treatment
Mwyngil Therapeutics is a new biotech startup pursuing obesity treatment without GLP-1 targets, led by CEO Luba Greenwood, aiming to explore alternative mechanisms beyond the GLP-1–driven weight-loss wave.
Biotech Pulse: Tau Drugs, SSRI Tapers, and a Sanofi Wearable Win
A Readout newsletter recap notes buzz around daraxonrasib and tau therapies at major meetings, U.S. HHS is drafting guidance on tapering SSRIs, and the FDA cleared Sanofi’s wearable injector for a myeloma drug.
Pancreatic cancer braces for post-daraxonrasib era
At a European pancreatic cancer conference, experts hailed the anticipated launch of daraxonrasib as a potential milestone that could reshape care, echoing the impact of checkpoint inhibitors. But they warned the new therapy would bring opportunities and challenges—from access and pricing to real-world effectiveness and how it will be integrated into standard treatment and combination strategies as data emerge.
AI uncovers the data behind Novo's Parkinson's stem-cell program
After Novo Nordisk shuttered its cell-therapy unit, Cellular Intelligence hired neurologist Nuno Mendonça to perform due diligence on Novo’s Parkinson’s stem-cell program. The data trail—drawn from Lund University research and Nature Medicine–published work on dopamine-neuron therapies—appears to have driven the AI-driven assessment that led the firm to pursue Novo’s asset.
US Biotech Firms Go Quiet as China Emerges as a Rival
As Chinese biotech competition intensifies, US drugmakers are increasingly secretive and cautious about intellectual property and development strategies, fueling a shift toward more guarded operations in areas like AI-driven drug discovery and next-gen therapies.
AZ and Ionis' heart-disease drug flops in pivotal ATTR-CM trial
AstraZeneca and Ionis reported that their heart-disease drug Wainua failed to outperform placebo in a pivotal trial for ATTR-CM (transthyretin-mediated amyloid cardiomyopathy), not reducing cardiovascular death or clinical events, and the news pushed both companies' shares down in premarket trading.