News

Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.

Already, NG-641 has shown encouraging preliminary safety and tolerability results in the ongoing phase 1a dose-escalation STAR trial (NCT04053283), which is a first-in-human study for the gene therapy.

Erandi De Silva, PhD, co-founder and senior vice president of product development at Forge Biologics, discusses the dual approach of her company in manufacturing and also developing adeno-associated viral (AAV) -based gene therapies, including FBX-101 for Krabbe disease.

Peter J. McAllister, MD, FAAN, presented promising data from the phase 2 STEMTRA trial at AAN 2022.

While the lower IV dose was generally well-tolerated, the higher dose yielded cases of serious cytokine release syndrome.

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

The decision was based on findings from the phase 1 TRANSCEND NHL 001 clinical trial.

The phase 1, investigator-initiated trial continues to recruit participants with B-ALL.

The novel regimen from BioNTech demonstrated encouraging results in patients with testicular or ovarian cancer.

AFM13 is currently also being evaluated on its own in lymphoma in a phase 2 registrational study.

The first in-human phase 1 clinical trial is being conducted in Japan.

Review top news and interview highlights from the week ending April 8, 2022.

Iovance plans to complete BLA submission for lifileucel by August 2022.

The phase 3 registration study is evaluating AB-205's efficacy in treating damaged stem cell niches.

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

The investigational therapy leverages an advanced overnight nonviral gene delivery manufacturing process that may help it overcome existing treatment limitations.

The biotechnology company previously pulled out of Europe after encountering payor challenges for their gene therapies.

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.

Review top news and interview highlights from the week ending March 25, 2022.

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.