News

The same therapy by Capricor Therapeutics has demonstrated efficacy in Duchenne muscular dystrophy.

The latest data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile.

The gene therapy ASC618 has received European and US designations and opinions that could help expedite development for hemophilia A.

The FDA has approved second-line axicabtagene ciloleucel as a treatment for adult patients with large B-cell lymphoma following frontline chemoimmunotherapy.

Review top news and interview highlights from the week ending April 1, 2022.

Positive interim results from a phase 3 study presented at AAD 2022 bolster recently published phase 2 data.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Allogene’s clinical hold on their AlloCAR T candidates was previously lifted in January 2022.

Research presented at the EBMT meeting suggests cell expansion may be a good biomarker of both response and outcomes.

A prespecified interim analysis from the phase 3 TRANSFORM study showed an event-free survival benefit with the second-line therapy.

A2 Biotherapeutics is developing CEA- and MSLN- targeted Tmod CAR T-cell therapies.

The professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Seven patients from the trial continued to be followed-up as of the data cut-off date.

Michael Singer, MD, PhD, cofounder and chief scientific officer of Cartesian Therapeutics, discussed the company’s lead candidate, Descartes-08.

The company’s lead cell therapy candidate, OpRegen, has demonstrated efficacy in dry AMD.

Complete results from the phase 3 GENEr8 study were published in the New England Journal of Medicine.

The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.

A sister study of AMT-130 in Europe dosed its first patients in February.

Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.

The company recently received IND clearance for a solid tumor cell therapy.

Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

Olaparib is now the first early-line therapy targeting BRCA-mutations in breast cancer.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.

Half the patients in the high-dose cohort of a phase 1a/2b trial achieved complete responses.

Review top news and interview highlights from the week ending March 18, 2022.

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.