Victoria Johnson

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

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

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

Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.

The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.

The gene therapy is currently under FDA review for the treatment of β-thalassemia across genotypes.

Results from the final analysis of the HOPE-B trial were presented at the 2022 EAHAD meeting.

No dose-limiting toxicities or treatment-emergent adverse events have been reported.

The hold was prompted by the second development of a serious AE of thrombotic microangiopathy in a patient in the younger age group.

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

SwanBio previously presented positive preclinical data at the ESGCT 2021 meeting.

The positive news is countered by a report of a patient death in Taysha’s gene therapy program for GM2 gangliosidosis.

Exciting new therapies are on the horizon for inherited retinal disease. We’ve compiled a quick snapshot of promising investigative therapies.

The latest hold on MB-207 follows a similar hold on another XSCID therapy, MB-107.

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

The SURPASS-2 trial will run parallel to the ongoing SURPASS trial.

The BASECAMP-1 study is identifying patients for future use of Tmod CAR T-cell therapies.

The FDA and Gamida Cell have reached a consensus on the comparability of omidubicel manufactured at different sites.

Data from the phase 2 QUILT-88 study were presented at the 2022 ASCO GI symposium.

Carisma Therapeutics and Moderna are also collaborating to develop CAR-M cell therapies.

Further details on the KEYNOTE-B79 trial were presented at the 2022 ASCO GI Symposium.

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

The phase 1/2 ARYA-2 trial will be run in parallel with the similar phase 1/2 ARYA-1 and ARYA-3 trials.

Eli-cel remains on clinical hold in cerebral adrenoleukodystrophy after a report of persistent anemia.

The first patient with Fabry disease dosed with FLT190 2 years ago continues to have elevated, sustained α-Gal A.

Cedrik Britten, MD, chief medical officer, Immatics, discussed the advantages of IMA203 over other cell therapies.

Details on the phase 1/2 CEDAR trial were presented at the 2021 ASH meeting.

Exciting new therapies for sickle cell disease are on the horizon. We explored some of the most promising investigational therapies in recognition of National Blood Donor Month.

Six of 7 patients reported improvements in dry mouth after treatment with AAV-hAQP1.

Many cell and gene therapy companies are initiating new programs and collaborations with the start of the new year.