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The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.

The director of the Mass General Brigham Gene and Cell Therapy Institute discussed the history and future of gene therapy in cardiology at the American Heart Association’s Scientific Sessions 2023.

One serious AE of myocardial infarction may have been related to VERVE-101 therapy while other AEs of fatal cardiac arrest and tachycardia were determined to be unrelated to the therapy.

Milind Desai, MD, MBA, an investigator on the MyPeak-1 trial and the director of the Hypertrophic Cardiomyopathy Center and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic, discussed Tenaya Therapeutics’ TN-201.

The trial will follow a phase 1/2/3 design and will evaluate GS-100 in pediatric patients with NGLY1 deficiency.

AVB-101 was simultaneously granted fast track designation by the agency. The company plans initiate US activities in 2024.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed unmet needs that remain, chiefly concerning widening the criteria for therapies and trials.

The median progression-free survival was 11 months and the SCG101 was well-tolerated.

Ben Creelan, MD, a medical oncologist at Moffitt Cancer Center, discussed several different approaches to cell therapy that could lead to new treatment options in the field.

Among 20 patients who received MDR-101, 12 have completed participation in the trial and have not taken their immunosuppression therapy for 2 years.

The therapy was previously granted priority review in May 2023.

The medical oncologist at Moffitt Cancer Center discussed 3 different approaches to cell therapy that could lead to new treatment options in the field.

Microsatellite instability (MSI) tumors are frequently found in people with Lynch syndrome, one of the most common hereditary colorectal cancers.

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

BRL Medicine is also enrolling a phase 1/2 multicenter trial evaluating BRL-201 at lower dose ranges.

The chief scientific officer of CureDuchenne discussed research he is looking forward to seeing in 2024.

The BASECAMP-1 screening study has now identified 28 patients whose solid tumors are positive for HLA-A*02 LOH.

David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, discussed findings from several preclinical studies that could help push engineered B-cell therapies towards clinical trials.

The chief scientific officer at Omega Therapeutics discussed milestones Omega Therapeutics is looking forward to in the next year.

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.