Tag

Gene Therapy

All articles tagged with #gene therapy

biotech3 days ago•2 min saved

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

health6 days ago•47 min saved

Sister's terminal diagnosis paves way for brother's life-saving gene therapy

A 5-year-old girl is diagnosed with metachromatic leukodystrophy, a rare genetic disease; her diagnosis leads doctors to test her younger brother, who is found to have the same condition and is now set to begin a $4 million gene therapy that could save his life, while the sister's decline underscores the emotional toll on the family.

via Yahoo|

#anna-somers #gene-therapy #health

health6 days ago•37 min saved

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

health11 days ago•106 min saved

Gene Therapy Breakthrough Brings Hope to Black Americans Living With Sickle Cell

A Louisiana man became the state’s first functionally cured sickle cell patient via 2023 FDA-approved gene therapies Casgevy and Lyfgenia; while these treatments hold promise for tens of thousands who are Black or African American, their high cost (around $2–$3 million) and limited treatment centers create access barriers, highlighting the need for broader insurance coverage, expanded care in underserved communities, and thorough discussion of risks like infertility and long‑term cancer risk.

via MS NOW|

#black-health #fda-approvals #gene-therapy

technology13 days ago•10 min saved

Epigenetic editing goes from concept to clinic in biotech startups

A new wave of biotech startups is pursuing targeted epigenetic editing—modifying DNA-associated chemical markers to turn genes on or off—aiming to treat diseases from muscular dystrophy to hepatitis B; companies like Epicrispr and nChroma are leading with dead-Cas9/Cas12F tools and refined methyltransferases, reporting preclinical success and early human dosing, while researchers caution that long‑term safety and off‑target effects remain critical as this approach moves toward the clinic.

via Nature|

#biotechnology-startups #crispr #epigenetic-editing

science14 days ago•47 min saved

First-in-Human Gene Therapy Aims to Rewind Cellular Aging in Glaucoma

A Life Biosciences Phase 1 trial is testing ER-100, a gene therapy designed to reset the aging clock in eye cells using OSK (OCT4, SOX2, KLF4) to potentially restore vision in open-angle glaucoma. Early preclinical data suggest epigenetic restoration can improve cellular function, but the approach carries safety risks, including cancer, and it remains uncertain whether translating this to humans will succeed or be safe.

via Yahoo|

#epigenetic-restoration #gene-therapy #glaucoma

science15 days ago•17 min saved

Base editing embryos renews designer-baby debate

Base editing, a next-generation CRISPR tool, has been used to precisely modify DNA in human embryos by nicking only one DNA strand. The development marks a major technical advance but renews ethical and safety concerns about applying gene editing to human reproduction and the possibility of ‘designer babies’.

via The Washington Post|

#biotechnology #designer-babies #gene-therapy

health18 days ago•2 min saved

FDA reopens review of Regenxbio's Hunter syndrome gene therapy

The FDA is reconsidering Regenxbio’s experimental gene therapy for mucopolysaccharidosis type II (Hunter syndrome) after previously rejecting it, part of a series of recent agency reversals amid leadership changes; the case follows another reversal where UniQure moved to submit a Huntington’s disease gene therapy previously spurned by the agency.

via statnews.com|

#fda #gene-therapy #health

technology27 days ago•11 min saved

The Pacemaker Patch: An External Ultrasound Approach to Pacing the Heart

Researchers envision a chest-mounted patch that uses high-frequency ultrasound to pace the heart, made possible by a gene-therapy step that makes heart cells produce a sound-sensitive protein; the system would pair with a pocket-sized data/power module and has shown responses in rats, pigs, and human heart cells. While intriguing as a potential alternative to implanted pacemakers, it remains experimental, with safety, regulatory, and practicality questions to resolve before clinical use.

via Hackaday|

#bioengineering #gene-therapy #medical-hacks

science27 days ago•6 min saved

Ancient Giant Scorpion, Whale Graveyard, and Breakthroughs in Aging Science

Science roundup covers the giant ancient scorpion Praearcturus gigas, a world-first aging-reversal gene therapy in human trials (high risk), a lab study simulating nuclear fallout that reveals surprising cesium behavior, a dementia patient briefly regaining speech after a psilocybin dose, and a giant whale graveyard at 7,000 meters depth that reshapes understanding of whale-fall ecosystems and cetacean evolution.

via ScienceAlert|

#aging #dementia #gene-therapy

science1 month ago•7 min saved

Eye-Aging Reprogramming Trial Doses First Patient in Vision Rescue Study

Life Biosciences dosed the first patient in a small, safety-focused trial of ER-100, a gene therapy designed to reset epigenetic marks in retinal ganglion cells to reverse aging-like changes and potentially restore vision in open-angle glaucoma or NAION. Delivered by a virus and activated by a specific antibiotic, the study will enroll up to 18 participants and follow them for years, attracting both optimism about aging biology and warnings about safety risks and uncertain efficacy.

via ScienceAlert|

#aging #er-100 #gene-therapy

health1 month ago•16 min saved

First Patient Dosed in Eye Aging Reversal Trial

Life Biosciences has dosed the first patient in an FDA-cleared trial of ER-100, a gene-therapy approach to reverse age-related degeneration of retinal neurons in glaucoma by reprogramming cells with Yamanaka factors activated by doxycycline for eight weeks. The work, building on Harvard's David Sinclair research, aims to rejuvenate vision but faces expert caution about potential runaway cell growth or cancer from cellular reprogramming.

via Gizmodo|

#aging #biotechnology #eye-health

health-and-wellness1 month ago•15 min saved

Israeli infant receives pioneering brain gene therapy for WOREE syndrome

An eight‑month‑old in Israel became the world’s first patient to receive an experimental brain gene‑replacement therapy aimed at restoring WWOX function to treat WOREE syndrome, delivered directly into the brain via an AAV9 vector at Schneider Children’s Medical Center. The procedure followed years of research led by Prof. Rami Aqeilan and supported by Mahzi Therapeutics; one month after treatment the baby was stable, had no seizures, and was discharged, but long‑term safety and efficacy require ongoing follow‑up.

via The Jerusalem Post|

#aav9 #gene-therapy #health-and-wellness

technology1 month ago•23 min saved

MIT's Wearable Ultrasound Patch Paces the Heart Without Surgery

MIT researchers have created a chest-worn hydrogel patch that noninvasively paces the heart by enabling engineered heart cells to respond to ultrasound via a gene-therapy primer—a technique called sonogenetics. In tests on rats and pig hearts, the patch restored steady rhythms during daily activity, pointing toward a potential noninvasive alternative to implanted pacemakers and, with future closed‑loop sensing, a fully automated pacing system.

via ZME Science|

#gene-therapy #pacemaker #sonogenetics

health1 month ago•3 min saved

FDA Unveils Draft Route to Speed Gene and Cell Therapies to Patients

The FDA issued a draft guidance showing how sponsors can reuse existing public data—such as CMC, nonclinical and clinical results—to streamline regulatory submissions for somatic genome-editing therapies, with the goal of accelerating access for patients with rare or life-threatening diseases while maintaining safety and efficacy; sponsors are encouraged to engage early (e.g., INTERACT) and submit comments within 90 days.

via fda.gov|

#cell-therapy #development-acceleration #fda