News

In a late-breaking presentation at ASGCT 2022, investigators shared preclinical results of a gene-editing strategy that seeks to reactivate developmentally silenced fetal hemoglobin (HbF, α2γ2) in order to replace defective sickle hemoglobin (HbS, α2βS2).

Study sponsor Pfizer recently announced the launch of a phase 3 trial in patients with Duchenne muscular dystrophy.

Interim data from the phase 2 Skyline trial demonstrated a 62.5% response rate.

Further data from Mustang Bio’s phase 1/2 trial will be presented at the EHA 2022 Congress.

Principal investigator Barry Greenberg, MD, FHFSA, shares extended results from the phase 1 trial and also preliminary pediatric safety results in a presentation at ASGCT 2022.

The 3 patients in cohort 1 have exhibited clinically meaningful improvements and no safety concerns. Follow-up for the 5 patients in cohort 2 is ongoing.

At ASCGT 2022, Italian investigators report updated safety and efficacy data from a phase 1/2 trial, in which all evaluable subjects achieved platelet transfusion independence and reported improved median platelet counts.

Review top news and interview highlights from the week ending May 13, 2022.

The locally injected gene therapy product is the subject of an ongoing phase 2a clinical trial.

The investigational therapy is also currently being evaluated in a clinical trial for sickle cell disease.

Caribou will present the initial data at the upcoming European Hematology Association (EHA) meeting in June.

Late-breaking interim data were presented at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders.

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

The cell therapy is the first to come out of the company’s proprietary ARC-SparX platform.

Alexander I. Spira, MD, PhD, FACP, research institute director with Virginia Cancer Specialists Research Institute, discusses the examination of RTX-240 in a phase 1 trial (NCT04372706) with solid tumors, presented recently at the 2022 AACR Annual Meeting.

The trial was set to begin patient dosing in the first half of this year.

Tim Lu, MD, PhD, CEO and co-founder of gene circuit company Senti Bio, joins CGTL to discuss his company's approach to creating "smarter" therapies, including its CAR-NK platform.

The hold was originally placed after reports of 2 serious adverse events in pediatric patients.

Marker Therapeutics is taking a multi-antigen approach to developing next-generation T-cell therapies.

Review top news and interview highlights from the week ending May 6, 2022.

Investigators presented up to 2 years of post-marketing data on the first-ever gene therapy.

Radek Špíšek, PhD, co-founder and chief executive officer of SOTIO Global, and Geoffrey Hodge, chief executive officer of SOTIO BioTech US, join CGTL to discuss its 3 technology platforms and how they are leveraging them to attack the solid tumor micro-environment.

Updated data at ARVO show that AGTC-501 had sustained efficacy and safety at 18 months for patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Veeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discusses advanced therapies for retinal diseases.

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

Although the agency has cleared the trial to restart, Pfizer will extend the voluntary hold on dosing new patients while the company talks through conditions of the restart.

Enrollment is ongoing in the phase 1 CEDAR trial, which plans to dose the first patient later in 2022.

The AAV9 gene therapy is currently being evaluated in a clinical trial conducted by NINDS.

New data from cohort 4 showed outer retinal restoration following treatment with OpRegen.

The post-hoc analysis of the OPTIC trial explored the effects of neutralizing antibodies on patients treated with ADVM-002.