Biotech & Medicine
185 articles RSS
Two teams publish DNA-guided CRISPR-Cas12 systems in Nature Biotechnology, inverting the RNA-guide paradigm for RNA diagnostics and antivirals
Researchers at the University of Florida and the Hong Kong University of Science and Technology each report a DNA-guided CRISPR-Cas12 platform that targets RNA, reducing off-target effects and enabling attomolar-level pathogen detection.
United Therapeutics Wins FDA Clearance for EXPRESS, the First Pig-Heart Xenotransplant Trial Designed to Support a BLA
The FDA cleared an IND for UHeart, a 10-gene-edited pig heart, opening a single-center phase 1/2/3 trial that will enroll up to two patients with end-stage heart failure before any expansion.
NEJM Publishes Phase 1/2 Data for Daraxonrasib as FDA Authorizes Expanded Access for Metastatic Pancreatic Cancer
NEJM publishes Phase 1/2 data for oral RAS(ON) inhibitor daraxonrasib (~30% ORR, 90% disease control in pretreated patients); FDA issues 'safe to proceed' for EAP on May 1, now available for eligible metastatic PDAC patients.
RNA-Triggered CRISPR System Selectively Kills Cancer and Viral Cells by Shredding Their DNA, Researchers Report in Nature
A University of Utah-led international team reports a new CRISPR nuclease, Cas12a2, that uses RNA guides to trigger widespread DNA shredding and cell death specifically in diseased cells such as KRAS-mutant cancers and HPV-infected cells, while leaving healthy cells unharmed.
Regenxbio's RGX-202 Hits Primary Endpoint in Pivotal Duchenne Trial, Clearing Path to a 2027 Accelerated-Approval Filing
Regenxbio's gene therapy RGX-202 met the primary endpoint of its pivotal Phase III AFFINITY DUCHENNE trial, with 93% of participants reaching the microdystrophin expression threshold.
AstraZeneca's Eneboparatide Frees 31% of Hypoparathyroidism Patients From Calcium and Vitamin D at Week 24 in CALYPSO Phase III
AstraZeneca's CALYPSO Phase III hit its composite primary endpoint with 31.1% of eneboparatide patients normalizing serum calcium and dropping supplements at week 24 versus 5.9% on placebo, with benefits sustained through a 52-week extension.
FDA Expands argenx's VYVGART to All Adult Myasthenia Gravis Serotypes, Reaching Seronegative Patients for the First Time
The May 8 sBLA approval extends efgartigimod's label to MuSK, LRP4 and triple-seronegative gMG patients after the 119-patient ADAPT SERON trial hit its MG-ADL endpoint with a 3.35-point improvement at week 4 (p=0.0068).
DECISION Trial Misses Primary Endpoint as Pooled Meta-Analysis of 9,013 Patients Finds Digitalis Cuts Heart Failure Events 15 Percent
Low-dose digoxin failed its primary endpoint in 1,001 Dutch heart-failure patients, but a JAMA meta-analysis of DECISION, DIGIT-HF and the 1997 DIG trial published the same day finds digitalis cuts the combined risk of cardiovascular death or first worsening event by 15 percent.
CRISPR Therapeutics and Vertex post $43 million CASGEVY revenue in Q1 2026 as pediatric submission lands with FDA Priority Voucher
First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.
Takeda's TAK-881 Matches HYQVIA in Pivotal Phase 2/3 Trial While Halving the Infusion Volume for Primary Immunodeficiency Patients
A 20% subcutaneous immunoglobulin paired with hyaluronidase delivered the same antibody exposure as HYQVIA in half the volume, clearing Takeda's path to global filings in fiscal 2026.
FDA Approves Veppanu From Arvinas and Pfizer, the First Protein-Degrader Drug Cleared for Any Cancer
Vepdegestrant becomes the first PROTAC ever approved by the FDA, treating ESR1-mutated advanced breast cancer after one prior endocrine therapy.
Chiesi to Buy KalVista for $1.9 Billion in Its Biggest-Ever Deal, Wrapping the First Oral HAE Pill Into a Growing Rare Disease Portfolio
Italian drugmaker Chiesi will pay $27 a share for KalVista Pharmaceuticals to acquire Ekterly, the first oral on-demand treatment for hereditary angioedema, in a deal expected to close in the third quarter.