News

The new application is based on data from the phase 3 TRANSFORM trial.

The therapy has been well-tolerated in the 15 patients treated so far.

The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.

Researchers will use the model to study how lack of pigmentation affects RPE physiology and function.

The FDA cleared SBT-101's investigational new drug application earlier in February 2022.

The ophthalmologist from John A. Moran Eye Center discussed the close association between disease progression and uncovered risk alleles.

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

The company also announced that the phase 3 study of val-rox has completed enrollment.

Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.

Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.

The global head of research and development at Atara Biotherapeutics discussed the link between EBV and MS.

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

Only 1 patient has been dosed in the trial so far, which initiated in October 2021.

The FDA has approved LEXEO Therapeutics’ investigational new drug application.

The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed CAR T-cell therapies and safety signals of cilta-cel.

This designation follows a fast track designation and IND application approval for the agent.

Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.

The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.

Investigators did not find any clinical signs of rejection of the mismatched donor cells.

Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.

The translational immunologist and research professor at Perelman School of Medicine, University of Pennsylvania, discussed persistence of CD4+ CAR T-cells in CLL.

Interim safety data from the FORTIS study were presented at WORLDSympsoium.

The associate professor from Tufts University School of Medicine discussed the latest updates on the FOCUS trial.

Review top news and interview highlights from the week ending February 11, 2022.

AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.

Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.

The dual-mechanism of the therapy helps address both systemic and organ-specific deficits.

Clinical development will be stopped on the CNGA3 achromatopsia program as no clinical improvements were seen.