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CARVac RNA vaccine may amplify the persistence of the BNT211 CAR T-cell therapy.

The members of the AdComm were generally of the opinion that the off-target analyses conducted by Vertex were reasonably robust.

Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.

Despite the announcement, CEO Doug Ingram shared that the company is seeking a wider approval for delandistrogene moxeparvovec-rokl regardless of age or ambulatory status.

Other efforts to develop a gene therapy for OTOF congenital hearing loss include programs from Sensorion and Eli Lilly.

The assistant professor at MD Anderson Cancer Center discussed factors to consider when trying to treat patients with gastrointestinal cancers with cell therapy.

Patient preferences should be considered when selecting the optimal treatment regimen for patients with relapsed/refractory follicular lymphoma, as both CD19-directed CAR T-cell therapies and CD20-targeted bispecific antibodies can be efficacious.

The therapy’s PDUFA date is scheduled for December 8, 2023, and an Advisory Committee is set to meet on October 31, 2023, to discuss the treatment’s potential approval for sickle cell disease.

In observance of Epidermolysis Bullosa Awareness Week, the executive director of debra of America discussed how a combination of advanced and topical approaches may represent the future of EB treatment.

In light of the IND clearance, Tenaya now intends to go forward with plans for a phase 1b clinical trial (RIDGE-1) that will seek to recruit up to 15 adult patients.

One patient had their Cell Pouch removed after the donor islet cells were found to be contaminated with Candida albicans yeast.

Review top news and interview highlights from the week ending October 27, 2023.

In light of the IND clearance, the company is planning to carry out plans for a phase 1 clinical trial (AMpLify) for adult patients with r/r AML.

The combo, termed MB-109, consists of the MB-108 oncolytic virus and MB-101 CAR T-cell therapy.

In observance of World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed the changing landscape of care in this field.

The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.

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

In observance of Epidermolysis Bullosa Awareness Week, the debra of America representatives discussed unmet needs for the patient population and the approval of the first EB gene therapy.

AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.

Neurona Therapeutics also reported that this first patient cohort has completed enrollment in addition to the early efficacy data from the first 2 patients treated.

The professor in residence of pediatrics at University of California San Francisco discussed a potential long-term consequence of gene therapy and myeloablative conditioning.

The company expects that pivotal ATMOSPHERE and ASCENT trials will support a BLA filing in 2024 for wet AMD, while the AAVIATE trial evaluates the therapy in diabetic retinopathy.

The US IND application, on hold since November 2022, was cleared with the help of data from a clinical trial being conducted in New Zealand.

The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed the design of the MyPeak-1 clinical trial for TN-201 and the associated MyCLIMB natural history study.

October is National Breast Cancer Awareness Month, and thus serves as an additional moment to point to the progress made in cell and gene therapy development, as well as the lingering challenges.

The first patient will be treated with CTO1681 at UPMC Hillman Cancer Center.

Review top news and interview highlights from the week ending October 20, 2023.

The company will meet with the FDA again to discuss the future of the investigational cell therapy.

Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.