News

TN-201 uses an AAV vector-based approach to deliver a functional copy of the disease-targeted MYBPC3 gene to cardiomyocytes.

Patients with HNSCC had a median overall survival (OS) of 20.8 months and a 12-month OS rate of 59%.

UoM was previously collaborating on the development of AVR-RD-05 with AVROBIO, but regained the license to the HSC gene therapy following the company’s cessation of development on all programs.

For World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed how Nexcella’s NXC-201 could help address unmet needs.

In terms of safety, it was noted that repeated administration of CAR mRNA/LNP was well-tolerated by mice.

Review top news and interview highlights from the week ending Novmber 3, 2023.

CGTLive spoke to experts in the field about their experiences and impressions of B-VEC almost half a year after its approval for dystrophic epidermolysis bullosa.

Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.

The chief executive officer of Likarda discussed the company’s CSS technology and how it may reduce off-tissue effects.

The DSMB has cleared Atamyo to go forward with a planned dose of ATA-100 for the second cohort that is 3 times higher than the dose used for the first cohort.

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

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.