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Lilly’s one-dose gene editor cuts LDL by 62% in early trial
1 day ago•Source: statnews.com
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3.555 min•18 days ago
Bacteria-Inspired Peptide Disrupts Cancer Energy, Enhances Radiation Therapy
AurB, a bacteria-derived peptide, enters tumor cell mitochondria and blocks ATP production, starving cancer cells; in preclinical prostate cancer models, its activity is strongest when combined with radiation, slowing tumor growth and prompting plans for clinical trials.
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In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE
statnews.com•29 days ago
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Veradermics' oral hair loss drug shows strong regrowth in late-stage trial
statnews.com•1 month ago
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Biotech Start-Up Claims Lab-Grown Sperm Could Help Infertile Men (Awaiting Validation)
Utah-based Paterna Biosciences says it has found a way to coax sperm-forming stem cells from testicular tissue to become mature, functional sperm in a lab and used this sperm to create embryos; the work has not yet been peer-reviewed or independently verified, and the company plans larger studies to compare lab-made and natural sperm in men with infertility before attempting pregnancies; if validated, the approach could offer a biological option for some infertile men, but questions of cost and safety remain.
Lilly strikes $3.25B buyout of Kelonia Therapeutics in high-stakes biotech bet
Eli Lilly agreed to acquire Kelonia Therapeutics for $3.25 billion, with potential additional payments tied to clinical, regulatory, and commercial milestones. Kelonia—formerly Elcano Therapeutics—had survived on about $60 million over five years and nearly ran out of cash on three occasions, aided by Venrock’s early backing. The deal highlights the volatile, milestone-driven journey of biotech startups and the race to capitalize on cell-therapy advances.
Nektar's rezpeg shows promising hair regrowth in alopecia, moving toward Phase 3
Nektar Therapeutics reported that extended treatment with its experimental drug rezpeg led to meaningful hair growth in severe alopecia areata after one year, with 27% of participants reaching SALT Score 20 (80%+ scalp coverage). The response is comparable to or better than low-dose Olumiant, though not tested head-to-head, and Nektar plans to begin a Phase 3 trial later this year.
Boston scientists win big at Breakthrough Prizes, dubbed science's Oscars
Four Boston-area researchers were honored at the 2026 Breakthrough Prize ceremonies: Stuart Orkin and Lee Roberts received Breakthrough Prizes of $3 million each for gene editing and muon physics, while Dillon Brout (Boston University) and Shu-Heng Shao (MIT) were New Horizon Prize recipients, each $100,000, for cosmology and generalized symmetries in quantum field theory. The awards, called the 'Oscars of Science' and cofounded by Zuckerberg and Chan, celebrate long-term breakthroughs—from sickle cell gene-editing therapies to precision cosmology data and new physics ideas.
Experimental pancreatic cancer drug yields sizable survival gain in Phase 3, signaling potential rapid FDA approval
Revolution Medicines reported that its daily pill daraxonrasib produced a median overall survival of 13.2 months in a Phase 3 trial for advanced pancreatic adenocarcinoma, compared with 6.7 months for standard chemotherapy, a notable survival boost that could accelerate an FDA submission, with trial investigators like NYU Langone’s Paul Oberstein discussing the implications on The Readout Loud podcast.
Amyloid debates and peptide policy drive biotech news
FDA plans advisory panels to reconsider compounded peptides; a sweeping Cochrane review revives debates over amyloid-targeting Alzheimer’s drugs; Roche will run another Elevidys trial outside the U.S. as Sarepta markets it domestically; Lilly’s Foundayo GLP-1 pill shows non-inferiority to insulin glargine with a FDA submission planned by the end of Q2.
A GLP-1-free route to weight loss gains attention
A group of researchers behind GLP-1 obesity drugs propose an alternative: a high-dose molecule that activates GIP and glucagon receptors may achieve weight loss similar to GLP-1–targeting therapies, potentially with fewer side effects. The approach, funded by BlueWater Biosciences and described in Molecular Metabolism, is based on rodent and nonhuman primate data and has not yet been tested in humans, making findings uncertain and likely controversial for the field.
Off-the-shelf CAR-T shows potential to delay lymphoma relapse in interim Phase 3 results
Allogene Therapeutics reported interim Phase 3 data for its off-the-shelf CAR-T therapy cema-cel in B-cell lymphoma, showing 58% MRD negativity in treated patients versus 16% in those observed with standard care, a result that could delay or prevent cancer relapse in high-risk patients, though the data are preliminary.
Allogene’s off-the-shelf CAR-T shows early MRD clearance and outpatient potential in first-line LBCL (ALPHA3 interim)
Allogene Therapeutics reported interim futility data from the ALPHA3 trial in first-line consolidation LBCL, showing 58.3% MRD negativity with cemacabtagene ansegedleucel (Cema-Cel) vs 16.7% in the observation arm, a 41.6-point absolute difference that aligns with literature suggesting meaningful potential benefit. Day-45 plasma ctDNA decreased 97.7% in the Cema-Cel arm versus a 26.6% median increase in the observation arm. The regimen was generally well-tolerated with no CRS, ICANS, or GvHD and most patients managed outpatient; enrollment is ongoing with interim EFS expected in mid-2027 and primary EFS in mid-2028, and a potential BLA submission if results remain favorable. Community centers contributed about one-third of screening and infusions, underscoring broader access potential for an off-the-shelf CAR-T therapy.
Revolution Medicines' targeted pill delivers striking survival advantage in pancreatic cancer
In a trial for metastatic pancreatic cancer, Revolution Medicines' targeted pill daraxonrasib extended median overall survival to 13.2 months versus 6.7 months with chemotherapy, a dramatic near-doubling that experts described as very impressive.