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Gene Therapy

All articles tagged with #gene therapy

FDA hold stalls Regenxbio's RGX-121 refiling amid spine MRI findings
biotech1 day ago

FDA hold stalls Regenxbio's RGX-121 refiling amid spine MRI findings

The FDA has placed a clinical hold on Regenxbio’s RGX-121 due to asymptomatic spine MRI findings, derailing plans to refile for approval in MPS II (Hunter syndrome). The hold follows a prior safety signal in RGX-111 and expanded monitoring that identified five patients with likely benign spinal nodules; researchers will continue imaging and use FDA feedback to determine next steps, while Regenxbio forges ahead with RGX-202 and surabgene lomparvovec. Regenxbio’s stock declined in premarket trading.

via Fierce Biotech|
#biotech#clinical-hold#fda
A Family's $4 Million Bet on a Groundbreaking Gene Therapy
health8 days ago

A Family's $4 Million Bet on a Groundbreaking Gene Therapy

Jo Kaur describes how her family created a patient‑driven initiative to develop a first‑in‑human gene therapy for their son with Cockayne syndrome, raising about $4 million to bring the treatment from concept to clinic and weighing whether to proceed with the procedure; three months post‑treatment, their son is clinically stable and showing encouraging signs, though long‑term benefits are still uncertain and the family aims to secure FDA approval to treat other children.

via aol.com|
#clinical-trial#cockayne-syndrome#fda
Chemogenetic brain therapies enter the clinic as China leads DREADD trials
science11 days ago

Chemogenetic brain therapies enter the clinic as China leads DREADD trials

US researchers at a NIH BRAIN Initiative meeting were stunned to learn that seven Chinese trials are testing DREADDs—designer receptors activated by a designer drug—in humans to suppress targeted brain activity for conditions like epilepsy, Parkinson’s disease, and neuropathic pain. The approach uses viral delivery to express receptors in specific neurons; when the drug is administered, activity in those circuits diminishes. While potentially offering circuit-specific therapies with fewer off-target effects, gene-therapy vectors carry immune risks. A trigeminal neuropathic pain trial has the most potential, and success could open circuit-based neurotherapies for many neuropsychiatric diseases. The pace suggests China is leading in this frontier.

via Chemical & Engineering News|
#chemogenetics#clinical-trials#dreadds
Rare gene tweak speeds metabolism, keeping some people naturally lean
science19 days ago

Rare gene tweak speeds metabolism, keeping some people naturally lean

A study of genetic data from over a million people identified a rare FNIP1 gene mutation that turns off calorie-burning pathways, helping about 1 in 7,000 individuals stay lean with lower body fat and healthier metabolic markers. Mouse experiments suggest turning off FNIP1 could inspire future weight‑loss therapies, but risks exist and any treatment would be years away.

via New York Post|
#fnip1#gene-therapy#genetics
Breakthrough SHP protein could shield joints, slow osteoarthritis in early studies
health23 days ago

Breakthrough SHP protein could shield joints, slow osteoarthritis in early studies

South Korean researchers identify SHP (NR0B2) as a natural cartilage protector; in animal models, restoring SHP reduced joint damage and pain, and SHP gene delivery into damaged joints yielded lasting benefits, suggesting a potential approach to slow or prevent osteoarthritis—though human trials are needed and results are preclinical.

via Futura, le média qui explore le monde|
#cartilage#gene-therapy#health
Mei’s Brain Gene Therapy Tragedy Reopens Debate on Biotech Safety
health24 days ago

Mei’s Brain Gene Therapy Tragedy Reopens Debate on Biotech Safety

Mei, a six-year-old in Shanghai, received a brain-targeted CRISPR-based gene therapy for a rare neurodevelopmental disorder and died soon after the viral injection. Hospital records point to a severe immune/vascular reaction (thrombotic microangiopathy) as the likely cause, and investigators found preclinical toxicity signals were not adequately disclosed to the ethics board while the trial lacked proper public updates and dose-testing. The case underscores gaps in China’s clinical-research oversight and transparency amid intense biotech competition, reminding the field that patient safety must come before pioneering claims.

via futura-sciences.com|
#china#clinical-trials#ethics
Mei's death from experimental gene therapy prompts ethics questions in China
science26 days ago

Mei's death from experimental gene therapy prompts ethics questions in China

Six-year-old Mei with a Rett-like Snijders Blok-Campeau syndrome died in March 2025 after participating in an experimental gene-therapy trial in China; Science and Retraction Watch report the trial violated ethical and safety rules, with Mei's parents funding a base-editing approach delivered by a viral vector, raising urgent questions about oversight and safety in biotech research in China.

via Le Monde.fr|
#base-editing#china#ethics-in-biotech
Six-year-old's death after unproven gene therapy in China sparks accountability push
health1 month ago

Six-year-old's death after unproven gene therapy in China sparks accountability push

Mei, a 6-year-old girl in Shanghai, died within days of receiving an experimental, virus-based gene therapy for a nonlife-threatening genetic disorder; her parents paid about $860,000 for the treatment, which lacked robust ethics review and regulatory oversight, and monkey studies had flagged liver and kidney damage and immune risks before the procedure; officials attributed her death to thrombotic microangiopathy, and a Science/Retraction Watch investigation has heightened questions about China’s handling of experimental gene therapies and accountability for the researchers and institutions involved.

via Live Science|
#china#death#ethics
Boston Partners Launch Nonprofit Center to Speed Gene Therapies for Rare Diseases
health1 month ago

Boston Partners Launch Nonprofit Center to Speed Gene Therapies for Rare Diseases

Boston Children’s Hospital, the Broad Institute, and Jackson Laboratory are launching the Center for Therapeutic Genetics, a nonprofit collaboration to develop and share programmable gene-editing therapies for rare diseases and make them more scalable and accessible by building reusable platforms—models, manufacturing data, safety data, and clinical programs—so doctors can deliver these treatments like standard medical procedures; backed by ARPA-H with up to $160 million, the center will initially target pediatric genetic epilepsies and is in its founding phase, not yet accepting patient referrals.

via The Boston Globe|
#biotech#boston#gene-editing
Epigenetic editors tune gene activity without altering the DNA
technology1 month ago

Epigenetic editors tune gene activity without altering the DNA

Researchers are using CRISPR-based epigenome editors to add or remove chemical marks on DNA and histones, enabling precise, multi-site control of gene expression without changing the DNA sequence. This tunable approach can produce temporary or lasting effects and is being explored in basic research, crops, and therapies, with early clinical strides and notable animal data—for example, long-term suppression of PCSK9 in mice and substantial reductions in PCSK9 and LDL in monkeys—alongside companies pursuing hepatitis B therapies and CAR-T enhancements that couple epigenetic editing with gene editing.

via Nature|
#biotechnology#crispr#epigenetics
MeiraGTx lands up to $400M to accelerate commercialization of late-stage gene therapies
biotech1 month ago

MeiraGTx lands up to $400M to accelerate commercialization of late-stage gene therapies

MeiraGTx secured up to $400 million from Oberland Capital to push its late-stage gene therapies toward launch, anchored by an initial $125 million loan with low single-digit royalties and a $10 million equity stake. The deal also includes up to $50 million in milestones for FDA approvals (one per program for bota-vec and AAV2-hAQP1), up to $100 million more via mutual commitments, and a right for Oberland to buy an additional $15 million in equity. In addition, $50 million could follow a positive AQUAx2 phase 2 readout for dry mouth. Beyond bota-vec and AAV2-hAQP1, MeiraGTx is advancing AAV-GAD for Parkinson’s, with a 2029 follow-up study, and noted a 2025 Lilly license for AAV-AIPL1, highlighting a pipeline with several near-term regulatory milestones.

via Fierce Biotech|
#biotech#biotech-funding#commercialization
Sick 5-Year-Old Sparks Lifesaving Gene Therapy for Younger Brother
health1 month ago

Sick 5-Year-Old Sparks Lifesaving Gene Therapy for Younger Brother

A Long Beach, NY family faces the rapid decline of 5-year-old Anna Somers from metachromatic leukodystrophy (MLD), a rare genetic disease with no cure. Her diagnosis leads doctors to test her 2-year-old brother, Joey, who is also found to have MLD. With a gene therapy approved in 2024 costing about $4 million, Joey will begin treatment before symptoms appear, offering a chance to save his life even as Anna’s condition continues to deteriorate.

via Yahoo News Canada|
#anna-somers#childhood-dementia#gene-therapy