News

The company recently received IND clearance for a solid tumor cell therapy.

Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

Olaparib is now the first early-line therapy targeting BRCA-mutations in breast cancer.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.

Half the patients in the high-dose cohort of a phase 1a/2b trial achieved complete responses.

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

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.

Another MSC therapy being developed by Mesoblast demonstrated efficacy earlier in March.

The grant, given to Indiana University School of Medicine, will fund 3 major projects to improve different aspects of hemophilia gene therapy.

The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.

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

Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.

Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.

The new designation follows an IND clearance and fast track review in February 2022.

WU-CART-007 is the first clinical-stage allogeneic CAR therapy for T-cell malignancies in the US.

Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.

New data presented at MDA 2022 showcased positive motor and bulbar function data.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.

Further results will be presented at the American Urological Association Annual Meeting, May 13-16, 2022.

The global head of research and development at Atara Biotherapeutics discussed the future of MS research.

Christina Y. Weng, MD, MBA, Baylor College of Medicine, Houston, discussed therapies for macular degeneration.

The company is planning to initiate a phase 1 trial in the second half of 2022.

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

The therapy’s IND has been cleared and a first in-human trial is expected to begin later this year.

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

The first patient was dosed in the phase 1/2a trial earlier in March 2022.

Patients demonstrated a median reduction of 63.5% in CSF NfL levels 6 months after treatment.