Review top news and interview highlights from the week ending February 24, 2023.
Lysosomal Disorders
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Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.
The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.
Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.
FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the limited distribution model of arsa-cel in Europe.
No adverse events related to RP-L201 have been reported to date.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.
Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.
The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
NGN-401 is Neurogene’s second investigational gene therapy to enter clinical trials.
With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.
Review top news and interview highlights from the week ending January 20, 2023.
The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.
The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.
Review top news and interview highlights from the week ending January 13, 2023.
The assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.
