News

The trial design was informed by both patient and physician perspectives.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.

The pause follows a fatal serious adverse event in the first patient treated in the highest dose cohort.

The trial, sponsored by Mustang Bio, will continue to enroll patients and doses will be escalated to 300 million cells.

The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.

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 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.

Interim data from the SPOTLIGHT study were presented at the ASCO GU Symposium.

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.