Tag

Gene Therapy

All articles tagged with #gene therapy

science-and-biotech1 day ago•8 min saved

Nobel Prize for Optogenetics Ignites Race to Commercialize Vision and Brain Therapies

The 2026 Nobel Prize in Physiology or Medicine was awarded to Karl Deisseroth, Peter Hegemann, and Georg Nagel for developing optogenetics, a technique that uses light to control specific nerve cells. While the prize recognizes the foundational discovery of using light-sensitive proteins from algae to map brain circuits, the immediate commercial impact is concentrated in ophthalmology. Biotech firms are racing to bring optogenetic gene therapies for retinal blindness to market, with Nanoscope Therapeutics and GenSight Biologics advancing clinical trials. Deisseroth’s company, MapLight, is also leveraging the technology for psychiatric drug discovery, though its initial candidates have faced mixed results.

via Fierce Biotech|

#gene-therapy #karl-deisseroth #nobel-prize-2026

health-and-medicine3 days ago•0 min saved

Optogenetic Trial Shows Partial Vision Restoration in 10 Patients with Retinitis Pigmentosa

A new study published in the New England Journal of Medicine confirms that optogenetic gene therapy can safely restore partial vision in people with advanced retinitis pigmentosa. In a trial of 10 participants, six showed clinically meaningful improvements in light sensitivity, and some regained the ability to detect objects and navigate simple tasks using custom goggles. The therapy, which leverages Nobel Prize-winning technology, involves a single injection to make surviving ganglion cells light-sensitive. While results are limited to monochrome perception and do not yet restore facial recognition, researchers aim to achieve high-resolution vision within five to 10 years.

via The Guardian|

#gene-therapy #health-and-medicine #nobel-prize

health4 days ago•26 min saved

One-time gene therapy repairs damaged adult retinas in dogs, offering hope for human vision loss

A single-dose gene therapy has successfully repaired damaged retinal connections in adult dogs, offering hope for treating inherited vision loss in humans. The treatment, tested on whippets with a faulty CaBP4 gene, restored vision and neural structures previously thought impossible to repair in mammals.

via New York Post|

#cabp4 #gene-therapy #health

science-and-technology5 days ago•29 min saved

Engineered Avian Retrotransposons Achieve 60% Targeted Gene Integration in Human Cells

Researchers at the Beijing Institute of Genomics identified 159 new avian R2 retrotransposons by scanning 1,139 bird genomes. By engineering these elements, they created an all-RNA system that achieves up to 60% site-specific gene integration in human primary cells, offering a new tool for precise genome editing without double-strand DNA breaks.

via Nature|

#gene-therapy #genome-editing #molecular-biology

science6 days ago•3 min saved

Gene Therapy Restores Damaged Retinal Connections in Dogs, Offering Hope for Human Vision Loss

A single-dose gene therapy trial in whippet dogs has successfully halted and reversed vision loss caused by a faulty CaBP4 gene. The treatment not only improved sight but physically repaired damaged neural connections in the adult retina, challenging the belief that such repair is impossible in mammals.

via ScienceAlert|

#cabp4-gene #gene-therapy #molecular-therapy-advances

science9 days ago•7 min saved

Beyond CRISPR: New Tools Enable Large-Scale Gene Replacement

New gene-editing technologies are emerging to overcome the limitations of CRISPR-Cas9, specifically its inability to efficiently insert large DNA segments. Techniques like PASTE, evoCAST, and recombineering allow researchers to replace entire faulty genes or engineer complex cellular circuits, addressing diseases with multiple genetic variants. While these methods offer broader therapeutic potential, they face significant challenges in delivery efficiency and complexity. The global CRISPR market is projected to grow at 16% annually, driven by these advancements and expanding clinical trials.

via Nature|

#biotechnology #crispr #gene-therapy

biotechnology11 days ago•0 min saved

uniQure Stock Plunges 41% as Huntington’s Gene Therapy Misses Key 48-Month Endpoint

uniQure shares dropped 41% after its Huntington’s disease gene therapy, AMT-130, failed to reach statistical significance on its primary endpoint at 48 months. While the composite Unified Huntington’s Disease Rating Scale (cUHDRS) showed only a 44% slowing of progression, the secondary Total Functional Capacity (TFC) endpoint remained strong at 61%. Analysts and executives argue that methodological flaws in the control group and the stability of functional outcomes support the drug’s path to accelerated FDA approval, despite the initial market sell-off.

via Fierce Biotech|

#amt-130 #biotechnology #fda-approval

biotechnology15 days ago•0 min saved

Endpoints News Unveils 2026 Top 11 Biotech Startups Amid Industry Shifts

Endpoints News has released its 2026 Endpoints 11 list, highlighting 11 private biotech startups selected from over 150 nominations. The awards, announced at a Boston gala, recognize companies making significant bets in gene therapy, oncology, and AI, reflecting a biotech sector that is recovering financially but facing new structural uncertainties.

via Endpoints News|

#ai-in-biotech #biotech-startups #biotechnology

science-and-health15 days ago•4 min saved

Epigenetic Therapy Silences Hepatitis B Virus in Early Trials

A new epigenetic therapy, CRMA-1001, has shown promise in silencing the hepatitis B virus (HBV) by adding chemical tags to viral DNA rather than cutting it. Developed by nChroma Bio and Italian researchers, the treatment targets both free-floating and integrated HBV DNA in the liver. Preclinical tests in human cells, mice, and monkeys yielded strong results with minimal side effects. A clinical trial has now begun in Hong Kong and New Zealand, with the first patient treated in January 2026. This approach aims to offer a potential cure for the 250 million people globally affected by chronic HBV, avoiding the cancer risks associated with traditional gene-editing methods.

via Nature|

#clinical-trials #crispr #epigenetics

health19 days ago•409 min saved

Gene therapy eases daily cornstarch burden for teen with rare metabolic disease

Thirteen-year-old Trey McGowan has a rare glycogen storage disease Type 1a (von Gierke) that requires uncooked cornstarch every few hours to prevent dangerous lows; after FDA-approved gene therapy (Genglycos) his daily cornstarch intake dropped and feeds are now six per day, easing the family’s anxiety and keeping him on a life-sustaining regimen that will always include cornstarch.

via TODAY.com|

#cornstarch-therapy #fda-approval #gene-therapy

health22 days ago•2 min saved

FDA approves first gene therapy for Sanfilippo syndrome, a rare childhood neurodegenerative disease

The FDA approved Ultragenyx’s gene therapy Fayuvi as the first treatment for Sanfilippo syndrome type A, a rare pediatric neurodegenerative disease, with price not disclosed.

via STAT|

#fayuvi #fda-approval #gene-therapy

health1 month ago•5 min saved

In-body CAR-T therapy shows early promise against multiple sclerosis

A small NEJM trial reports that a single intravenous injection of a lentiviral vector instructs the body to generate CAR-T cells in vivo, which then deplete B cells and autoantibodies and correlate with improved motor and cognitive function, reduced fatigue, and lower inflammation in people with multiple sclerosis and other autoimmune diseases over about six months. This supports the concept of in‑vivo CAR-T therapy as a cheaper, faster approach, but efficacy must be confirmed in more participants.

via Nature|

#autoimmune-diseases #car-t-therapy #gene-therapy

science1 month ago•0 min saved

China CRISPR Deaths Prompt Fresh Pushback on Viral Gene Therapy

The deaths of two children in China after receiving CRISPR-based therapies have renewed concerns among scientists about the safety and ethics of delivering gene-editing medicines with viral vectors, prompting renewed calls in the US for tighter standards and oversight.

via Endpoints News|

#bioethics #china #crispr

science1 month ago•3 min saved

Inhalable tRNA Therapy Reverses CF Defect, Paving Way for Other Genetic Diseases

University of Toronto researchers engineered an inhalable, modified tRNA that can read through premature stop codons in CFTR mRNA, restoring CFTR protein in mice and persisting in lung tissue for up to 40 days; in patient-derived organoids, tRNA plus the CF drug Trikafta restored function where the drug alone failed. The study suggests tRNA therapies may complement mRNA approaches and could be extended to Duchenne muscular dystrophy and Rett syndrome, with plans to move toward clinical trials in 2–3 years.

via Fierce Biotech|

#cystic-fibrosis #gene-therapy #inhalable-rna

biotech1 month ago•2 min saved

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