News

BioMarin plans to resubmit the biologics license application for val-rox to the FDA by the end of September 2022.

iECURE and the University of Pennsylvania Gene Therapy Program have collaborated to develop the GTP-506 program.

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

The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed requirements for administering beti-cel.

Matthew J. Frigault, MD, discussed safety and efficacy findings on CART-ddBCMA.

At a median follow-up of 24 months, the best ORR achieved was 77.6% among 58 evaluable patients with large B-cell lymphoma treated with relma-cel.

Merit Cudkowicz, MD, discussed corrected data from a phase 3 trial of NurOwn that BrainStorm believes supports a BLA submission.

If approved, the company will initiate the first-in-human STAR-101 phase 1 clinical trial.

The senior vice president and head of medical affairs at Orca Bio discussed the initiation of the Precision-T phase 3 trial.

Review top news and interview highlights from the week ending August 19, 2022.

The company recently announced a dose escalation in the ACCLAIM-1 study.

The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed beti-cel's approval.

The pheEDIT clinical trial initiated in June 2022.

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

ZYNTEGLO is now the world's most expensive gene therapy at a $2.8 million price tag.

Tessa Therapeutics presented positive data from the phase 2 CHARIOT study of TT11 in December 2021.

Multiple cell and gene therapy companies have been granted patents for novel technology in 2022.

The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.

BrainStorm Cell Therapeutics has decided to submit a BLA for NurOwn in ALS following an erratum to a phase 3 trial.

A safety review committee approved the dose-escalation after reviewing the first cohort of patients.

Preclinical data showed a reduction of Gys1 mRNA and protein of more than 80% in multiple skeletal tissues.

GentiBio will use their proprietary regulatory T cell platform to develop the engineered therapies against multiple targets.

The oncologist from Children's Hospital of Philadelphia discussed long-term safety data with tisagenlecleucel in pediatric patients with acute lymphoblastic leukemia.

Review top news and interview highlights from the week ending August 12, 2022.

The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.

AstroRx previously demonstrated clinically meaningful reductions in the rate of disease progression.

PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.

A single patient will be dosed with CRD-THM-001 in an upcoming trial.