News

The FDA has extended the PDUFA target action date for Capricor Therapeutics' deramiocel BLA from August 22 to November 22, 2026, after the company submitted 24-month HOPE-3 open-label extension data supporting a refined indication focused on upper limb function.

The FDA has accepted for priority review GSK's sBLA for Jemperli (dostarlimab) in previously untreated stage II/III dMMR/MSI-H locally advanced rectal cancer, based on AZUR-1 data, with a PDUFA date of February 2027 and eligibility for the National Priority Voucher program.

Genentech is betting up to $2.3 billion on an obesity drug designed to build muscle while cutting fat, addressing a GLP-1 side effect.

Today’s BioPharm Brief covers promising early-stage lung cancer data from AbbVie, an NMPA-accepted NDA for Leads Biolabs’ opamtistomig in rare neuroendocrine cancer, and a first-in-human trial of a brain-penetrant bispecific T-cell engager for glioblastoma.

The company's first-in-class immune-boosting antibody could become the first approved treatment for a cancer with no existing options.

Amplitude Therapeutics has entered a strategic research collaboration and licensing agreement with Eli Lilly and Company to discover and develop trans-amplifying RNA (taRNA) vaccine candidates for infectious diseases with high unmet need, with Lilly holding an option to add up to two additional targets.

AbbVie's bispecific antibody posted a 90% response rate in squamous NSCLC, headlining the company's expanding lung cancer pipeline data.

Adaptin Bio has opened enrollment in a first-in-human Phase 1 trial of APTN-101, a bispecific T-cell engager built on the company's BRiTE platform designed to cross the blood-brain barrier and target EGFRvIII-positive glioblastoma. The trial, conducted with Duke University, will enroll up to 15 adult patients with WHO Grade IV malignant glioma.

A defensible AI quality framework links intended use, patient and product risk, evidence, human accountability, and lifecycle control, without forcing every use case into the same validation model.

Dr Eric von Hofe of CellxLife and Dr Keith L. Knutson of Mayo Clinic explain why manufacturing speed and cost will determine the scalability of personalized cancer vaccines.

Avexitide cut severe hypoglycemic events 55% in a pivotal PBH trial, positioning it to become the condition's first approved therapy.

Kirsten Detrick, chief representative, USA, at Basel Area Life Sciences Supercluster, discusses why emerging biopharmaceutical companies should incorporate market access and commercial considerations earlier and how Basel’s concentration of cross-functional talent can support product development and launch.

Regeneron Pharmaceuticals' garetosmab-grts has become the first approved therapy to shrink new bone lesions in FOP, cutting them by up to 94% as it targets the disease's root biology directly.

The FDA has approved Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy, to reduce cornstarch dependence in patients eight and older with GSDIa, the first approved treatment to target the disease's underlying genetic cause.

AbelZeta Pharma has reclaimed global rights to prizloncabtagene autoleucel from Janssen and secured FDA clearance to begin US trials, positioning the anti-CD20/CD19 bispecific CAR-T for patients who relapse after existing CAR-T therapies.

This week’s BioPharm Brief covers positive phase 3 melanoma results for Merck and Moderna’s individualized neoantigen therapy, a personalized cancer vaccine manufacturing platform entering clinical use, and a new brain-penetrant antibody approach for glioblastoma.

AI tools that extract information from unstructured medical records could help oncology practices identify eligible clinical trial patients, surface care gaps, and compare treatment outcomes across real-world cohorts, according to Shaalan Beg and Eron Kelly of ConcertAI.

The companies are targeting a protein on glioblastoma's treatment-resistant tumor stem cells, which could open a new path past the blood-brain barrier.

Real-time process analytics and digital manufacturing systems could generate the reliable data needed for artificial intelligence to deliver more predictive insights in biopharmaceutical manufacturing, according to Bryan Hassell, founder and CEO of Nirrin Technologies.

Merck and Moderna report that the personalized mRNA plus mAb cancer therapy cleared its first phase 3 hurdle, cutting recurrence risk beyond pembrolizumab alone.

NTx Bio's NTxscribe CORE platform is being used by Baylor College of Medicine to manufacture mRNA for DOC1021, Diakonos Oncology's personalized dendritic cell vaccine, now dosing patients in a Phase 1 trial for refractory melanoma.

Today’s BioPharm Brief looks at a long-acting anti-VEGF therapy entering clinical trials, the first commercial use of imlifidase in Australia for kidney transplantation, and FDA Fast Track designation for a platelet-derived biologic for chronic sciatica.

CellxLife CEO Dr Eric von Hofe and Mayo Clinic immunology professor Dr Keith L. Knutson explain how dendritic cell vaccines generate long-term immune memory to prevent cancer recurrence.

Eyconis has dosed the first patients in OVERTURE, a phase 1b/2a first-in-human trial of EYC-0305, an investigational anti-VEGF antibody fragment designed for six-month or longer dosing intervals in wet age-related macular degeneration, marking the company's transition to clinical-stage status.

Australia's first commercial imlifidase transplant offers a path forward for patients whose antibodies had blocked a compatible kidney.

The FDA has granted Fast Track designation to C-1101, Consano Bio's investigational platelet-derived multi-protein biologic for chronic, painful lumbosacral radiculopathy, following the drug's recent IND clearance and as the company advances a Phase 1 trial evaluating the epidural injection.

Genprex moves its diabetes gene therapy toward the clinic, tapping a CGMP CDMO after preclinical models reversed hyperglycemia in mice.

Today’s BioPharm Brief looks at an oncology trial discontinuation, an AI-designed glioblastoma vaccine, and an expanded biosimilars collaboration.