Beam Therapeutics' Sickle Cell Therapy Clears Phase 1/2 Trials

The result represents a scientific milestone and positions Beam to submit a first-of-its-kind FDA application as soon as the end of the year.

Beam Therapeutics announced Tuesday that its base-edited sickle cell therapy succeeded in a clinical trial, meaning a third one-time sickle cell therapy may soon enter the market.

A phase 2 trial compares dual-targeted CAR T-cell therapy with R-GemOx as second-line treatment for patients with relapsed or refractory large B-cell lymphoma who are ineligible for transplantation.

Qureight, an end-to-end imaging company providing enterprise-grade imaging and precision endpoints for clinical trials for lung and heart disease, and Contineum Therapeutics, Inc. (Contineum), a …

In the Ez-PAVE trial, heart patients who began statin-ezetimibe therapy had similar three-year cardiovascular event rates to those who started with a statin alone, while lower LDL targets reduced …

A stroke survivor who quit rehab, a trial where 34 of 49 patients lost a lifelong fear, and what it means for any clinic still waiting.

Background:Persistent pain after extremity fracture requiring surgical repair is common and associated with long-term morbidity. We sought to determine whether app-based cognitive behavioural …

MDMA therapy for veterans PTSD advances as PharmAla Biotech signs a supply-and-data deal with Denmark's Aalborg University Hospital for the MIRROR study, trading discounted GMP MDMA for a perpetual …

CRISPR therapy CTX310 reduced triglycerides 63% in a Phase 1b trial for severe hypertriglyceridemia, pointing to a potential one-time treatment.

An Australian-developed therapy aimed at halting motor neurone disease progression and restoring function will advance to a national Phase 2 clinical trial.

A multicenter clinical trial evaluating chimeric antigen receptor-T cell therapy (CAR-T cell therapy) for stiff person syndrome (SPS), a rare autoimmune neurologic disorder, has produced promising …

A multicenter clinical trial evaluating chimeric antigen receptor-T cell therapy (CAR-T cell therapy) for stiff person syndrome (SPS), a rare autoimmune neurologic disorder, has produced promising …

CHICAGO — A monoclonal antibody that inhibits angiopoietin-like protein 4 was linked to triglyceride reduction at 12 weeks in adults with high triglycerides, researchers reported at the American …

A multicenter clinical trial evaluating chimeric antigen receptor-T cell therapy (CAR-T cell therapy) for stiff person syndrome (SPS), a rare autoimmune neurologic disorder, has produced promising …

A phase 2b clinical trial led by Mass General Brigham Heart and Vascular Institute researchers shows that a monoclonal antibody therapy called MAR001 can significantly reduce triglyceride levels …

Two case reports and several ongoing clinical trials highlight the potential of combination immune-modulating therapies to preserve beta-cell function in T1D. Medical News

Computer-designed protein therapeutics move from proof-of-concept to wet-lab validation and even clinical trials as the field heats up.

Children born small for gestational age maintained increases in mean annualized height velocity for up to 2 years with a once-weekly growth hormone therapy, according to data from the REAL8 and …

Sanofi halted development of an oral STAT6 degrader that had been under study in a Phase I first-in-human trial in Europe. STAT6, a key drug target in type 2 inflammation, is among the five targets …

ORLANDO — Scientific stakeholders and regulatory bodies alike will be essential to optimizing AI to help bring treatments like CAR T-cell therapy to the clinic, according to the keynote speaker at …

Scribe's CRISPR gene therapy held cholesterol down for two years from one shot — entering human trials the day pelacarsen failed its Phase 3 trial.

Trial led by Mount Sinai researcher could help improve long-term outcomes for these complex cases...

Patients with stable heart disease and abnormal stress tests who have invasive procedures early in their care do not live longer than those initially treated with drugs and lifestyle changes alone, …

CHICAGO — Ziltivekimab, a novel interleukin-6 inhibitor, lowered inflammatory markers but not major adverse CV events compared with placebo among adults with atherosclerotic CVD and chronic kidney …

Researchers from the PROFILE trial compared a top-down anti-TNF therapy strategy with a conventional step-up treatment strategy for patients with Crohn's disease.