Category REGENERATIVE MEDICINE

Simple Blood Protein Tests Predict Which Lymphoma Patients Are Most Likely to Have Poor CAR T Outcomes

International research team develops, validates approach for assessing and responding to elevated risk As new cancer treatments become available, some of the most important ongoing research must look at ways to optimize those new approaches so that more patients can benefit from groundbreaking therapies. In work newly published in Blood Cancer Discovery, a journal of the American Association

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Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant, the first to receive gene therapy in the United States for a genetic form of hearing loss AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF)

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The cause of Guillain-Barré syndrome elucidated

Guillain-Barré syndrome is a rare disease in which the immune system attacks the peripheral nerves. Those affected suffer from muscle weakness and paralysis. A research team led by ETH Zurich has now elucidated the mechanism of this autoimmune reaction. Patients with Guillain-Barré syndrome (GBS) suffer from a rare and diverse disorder of the peripheral nervous

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First-in-human clinical trial of CAR T cell therapy with new binding mechanism shows promising early responses

Improving CART19 function by targeting a membrane-proximal CD19 epitope with fast on- and off-rates. Early results for the Penn Medicine-developed AT101 presented at ASH Early results from a Phase I clinical trial of AT101, a new CAR T cell therapy that uses a distinct binding mechanism to target CD19, show a 100 percent complete response

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FDA Approves First CRISPR-based Gene Therapies to Treat Patients with Sickle Cell Disease

The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an

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Vitamin B12: a key player in cellular reprogramming and tissue regeneration

Researchers at IRB Barcelona reveal that vitamin B12 significantly boosts the efficiency of cellular reprogramming, thus holding promise for regenerative medicine. Vitamin B12 supplementation shows potential in speeding up tissue repair in a model of ulcerative colitis—an observation that points to potential new treatments for inflammatory diseases. The discovery has been published in the journal Nature

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Study Shows Stem Cell Transplant Significantly Improves Outcomes in Refractory Juvenile Systemic Sclerosis

New research at ACR Convergence 2023, the American College of Rheumatology’s (ACR) annual meeting, shows that patients with refractory juvenile systemic sclerosis improved significantly on nearly all measures for two years following autologous stem cell transplant (Abstract #L06). Juvenile-onset systemic sclerosis (jSSc), also called scleroderma, is a disfiguring autoimmune disorder marked by hardening of the skin

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VERVE-101: CRISPR-Based Gene Editing Therapy Shows Promise in Reducing LDL-C and PCSK9 Levels in Patients With HeFH

A single infusion of a CRISPR-based gene editing therapy significantly reduced LDL-C and PCSK9 levels in patients with heterozygous familial hypercholesterolemia (HeFH), based on findings from the VERVE-101 trial presented Nov. 12 at AHA 2023. In presenting the findings, Andrew Bellinger, MD, PhD, said they provide the first proof-of-concept for in vivo DNA base editing in humans.

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