Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2024-04-24

Dawn of CAR-T cell therapy in autoimmune diseases: Chinese Medical Journal review article highlights the potential and promise 

Credit: Chinese Medical JournalDaishi Tian from Huazhong University of Science and Technology Autoimmune disease (AID) refers to the condition in which the immune system identifies the body’s own cells and tissues as foreign, resulting in systemic inflammation. The immune system’s self-attack via autoreactive B and T immune cells and autoantibodies—antibodies against body’s own proteins—may present

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PIER MARIA FORNASARI
2024-03-19

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).  Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. It is caused by a

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No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2024-03-14

Mayo Clinic researchers find promise in new potential treatment for liver failure

A new drug has been shown to increase healing and regeneration of the liver after major surgery, according to a study published in the scientific journal Cell. Researchers hope that this could lead to more surgical options for patients diagnosed with advanced liver tumors and liver failure. “This research is significant because this is the first

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No Comments InREGENERATIVE MEDICINE
PIER MARIA FORNASARI
2024-03-14

A micro-fragmented collagen gel as a stem cell-assembling platform for critical limb ischemia repair

Critical limb ischemia is a condition in which the main blood vessels supplying blood to the legs are blocked, causing blood flow to gradually decrease as atherosclerosis progresses in the peripheral arteries. It is a severe form of peripheral artery disease that causes progressive closure of arteries in the lower extremity, leading to the necrosis

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No Comments InCELL THERAPY
PIER MARIA FORNASARI
2024-03-14

Preliminary Clinical Trial Results Show ‘Dramatic and Rapid’ Regression of Glioblastoma after Next Generation CAR-T Therapy

A collaborative project to bring the promise of cell therapy to patients with a deadly form of brain cancer has shown dramatic results among the first patients to receive the novel treatment. In a paper published today in The New England Journal of Medicine, researchers from the Mass General Cancer Center, a member of the Mass

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PIER MARIA FORNASARI
2024-02-06

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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PIER MARIA FORNASARI
2024-01-24

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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No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2024-01-20

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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PIER MARIA FORNASARI
2023-12-11

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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