Tag

Gene Therapy

All articles tagged with #gene therapy

health5 days ago•6 min saved

Knee-Worn Robot Enhances Leg Strength in Children with SMA Under Gene Therapy

A six-week study of a lightweight knee-mounted wearable robot providing isokinetic resistance improved knee function in six children with type 2 SMA who were receiving gene therapy, increasing quadriceps size by ~20% and more than doubling knee-extension strength, with MRI and nerve tests confirming muscular and neuromuscular gains and parents reporting easier daily movements at home; results suggest home-based rehab could augment gene therapy, though it’s not yet clear if the device offers unique advantages over other high-intensity exercise.

via Nature|

#gene-therapy #health #isokinetic-training

science8 days ago•1 min saved

AAV Gene Therapy Linked to Brain Tumor in Hurler Syndrome Case

A NEJM case report documents a young Hurler syndrome patient who received adeno-associated virus–delivered gene therapy and later developed a walnut-sized brain tumor believed to be caused by an unintended gene mutation; the tumor was removed, cognitive development progressed, highlighting rare but serious risks associated with gene therapies.

via Futurism|

#aav #brain-tumor #gene-therapy

science10 days ago•7 min saved

SHP Protein Emerges as Potential Breakthrough to Stall Osteoarthritis

Researchers have identified the SHP (NR0B2) protein as a key defender of cartilage whose levels fall as osteoarthritis progresses. Restoring SHP in animal models reduced cartilage damage, improved joint function, and dampened cartilage-degrading enzymes (MMP-3 and MMP-13) via the IKKβ/NF-κB pathway. Gene delivery of SHP into affected joints produced lasting benefits, suggesting a potential disease-modifying approach to slow or halt osteoarthritis.

via SciTechDaily|

#cartilage #gene-therapy #nf-kb-pathway

healthcare12 days ago•2 min saved

Regenxbio hits milestone in Duchenne gene therapy ahead of FDA filing

Regenxbio announced its experimental Duchenne muscular dystrophy gene therapy produced sufficient levels of a miniaturized muscle protein in a clinical trial, paving the way for an FDA submission and aiming to improve on Sarepta’s Elevidys in both efficacy and safety, amid ongoing safety concerns surrounding the competitor.

via statnews.com|

#biotechnology #clinical-trials #duchenne-muscular-dystrophy

health13 days ago•2 min saved

Toddler's brain tumor stirs caution over gene-therapy viruses

After a failed stem-cell transplant, a 13-month-old boy received a novel gene-therapy treatment and initially made progress, but a routine scan later revealed a golf-ball-sized brain tumor; scientists say the tumor may be linked to the gene-therapy viruses, highlighting the need to weigh life-saving benefits against rare but serious risks in such approaches.

via statnews.com|

#biotechnology #brain-tumor #gene-therapy

health1 month ago•24 min saved

FDA backs first gene therapy to treat a rare genetic deafness (Otarmeni) with free US access

The FDA approved Otarmeni, Regeneron’s gene therapy for OTOF-related deafness—the first gene therapy for this rare inherited hearing loss—delivered via a single cochlear injection to restore otoferlin production. In a 20-child trial, several participants showed hearing improvements, with five of 12 followed for 11 months reaching near-normal hearing. Regeneron will provide Otarmeni at no cost to US patients, and the therapy was fast-tracked under the Commissioner’s National Priority Voucher program. While promising, effects were generally safe, the condition affects about 50 US babies born each year, and this advance could pave the way for more gene therapies and potentially reduce reliance on cochlear implants.

via New York Post|

#fda-approval #gene-therapy #health

health1 month ago•2 min saved

FDA approves Otarmeni, first gene therapy for rare OTOF-related infant hearing loss

FDA approves Regeneron's Otarmeni, the first gene therapy for a rare OTOF-mutation–driven hearing loss in infants; in a trial, a single-dose inner-ear injection improved hearing in 16 of 20 children, with some able to detect whispers; the company will provide the drug for free to U.S. patients, while pricing outside the U.S. has not been determined since it is not approved there.

via NBC News|

#fda-approval #gene-therapy #health

healthcare1 month ago•13 min saved

FDA greenlights Otarmeni: first gene therapy to restore hearing in OTOF-related deafness, Regeneron to provide it free in the U.S.

The FDA granted accelerated approval for Otarmeni (lunsotogene parvec-cwha), a one-time gene therapy for severe-to-profound OTOF-related hearing loss, based on CHORD trial results showing 80% of participants reached a key hearing threshold at 24 weeks and 42% achieved normal hearing by 48 weeks; Regeneron will provide Otarmeni free to eligible U.S. patients as part of access programs, with continued confirmatory trials and safety monitoring ongoing.

via Regeneron|

#fda-accelerated-approval #gene-therapy #healthcare

business1 month ago•2 min saved

Regeneron trims prices for some Americans and pledges free hearing-loss gene therapy after FDA nod

Regeneron said it will lower U.S. drug prices for some Americans under President Trump’s pricing plan and will offer its first hearing-loss gene therapy for free to eligible patients after regulatory approval, while also securing a three-year tariff exemption; the therapy Otarmeni targets a rare hearing-loss condition and could reduce reliance on cochlear implants, with analysts signaling potential peak sales around $130 million.

via CNBC|

#business #drug-pricing #gene-therapy

health1 month ago•13 min saved

First-in-class Otarmeni gene therapy could restore hearing in rare OTOF-related loss, Regeneron to provide free US access

Regeneron’s Otarmeni (lunsotogene parvec-cwha) received FDA accelerated approval as the first in vivo gene therapy for OTOF-related severe-to-profound hearing loss, based on CHORD trial results showing 80% reached a PTA threshold of ≤70 dB HL at 24 weeks and 42% achieved normal hearing (≤25 dB HL) by 48 weeks. The one-time intracochlear infusion is intended for patients with biallelic OTOF variants, preserved outer hair cell function, and no prior cochlear implant in the treated ear, and Regeneron will provide Otarmeni at no cost to eligible U.S. patients. Safety signals include otitis media, dizziness and other procedure-related events; continued approval will depend on confirmatory CHORD data and site-specific eligibility constraints.

via Regeneron|

#fda #gene-therapy #health

health1 month ago•67 min saved

Gene therapy brings near-normal hearing to most with inherited deafness in largest trial

In the largest trial to date, a gene therapy delivering functional copies of the OTOF gene to the inner ear improved hearing in about 90% of participants (38 of 42) with congenital deafness caused by OTOF mutations, across eight sites in China. Improvements began within weeks and persisted for up to two years in many cases; some participants achieved normal conversational hearing, while a few showed no benefit. The therapy had no serious side effects, though some mild vertigo or inner-ear inflammation occurred, and younger patients tended to improve more. If further trials confirm long-term safety and broader applicability, the approach could offer a one‑and‑done alternative or complement to cochlear implants, with next steps including longer follow-up and dosing studies.

via Live Science|

#clinical-trial #congenital-deafness #gene-therapy

science1 month ago•25 min saved

Durable hearing gains from multicenter AAV1-hOTOF gene therapy for OTOF-related deafness

In a multicenter, single-arm trial across eight centers, 42 participants with autosomal recessive OTOF-related congenital deafness received AAV1-hOTOF at three doses. No dose-limiting toxicities were observed, and 90% of treated ears achieved hearing recovery with progressive, durable improvements in ABR/ASSR thresholds and speech perception up to 2.5 years. Younger participants showed greater gains, and baseline DPOAE strength and certain OTOF variants predicted better outcomes, supporting broad efficacy and safety of the therapy over up to 2.5 years.

via Nature|

#aav1 #congenital-deafness #gene-therapy

business1 month ago•6 min saved

Lilly to acquire Kelonia to push off-the-shelf in vivo CAR-T for multiple myeloma

Eli Lilly will acquire Kelonia Therapeutics for up to $7 billion in cash (upfront $3.25B) to gain Kelonia's in vivo gene delivery platform and KLN-1010, an in vivo anti-BCMA CAR-T therapy for multiple myeloma in Phase 1, with the aim of simplifying manufacturing and broadening access versus ex vivo CAR-T; the deal is expected to close in the second half of 2026, subject to regulatory approvals and milestone-driven payments.

via PR Newswire|

#biotechnology #business #car-t

science1 month ago•7 min saved

A Universal Regeneration Blueprint Could Restore Limbs

Scientists identify a conserved set of SP genes that govern limb regeneration across axolotls, zebrafish, and mice. By using a zebrafish-derived enhancer to drive a viral delivery of FGF8, they partially restore digit bone regrowth in mice, showing that reactivating the epidermal SP program can trigger regenerative pathways. While not yet applicable to full human limbs, this work provides a foundational gene-therapy approach that could complement bioengineered scaffolds and stem-cell strategies in the long term, moving toward true limb restoration rather than prosthetics.

via Neuroscience News|

#cross-species #fgf8 #gene-therapy

science1 month ago•3 min saved

Sight Restored: Gene Therapy Breakthrough Wins Major Science Prize

A married couple and investigator Katherine High share a $3 million Breakthrough Prize for Luxturna, the gene therapy approved in 2017 that restores vision in people with Leber congenital amaurosis by delivering a working RPE65 gene to retinal cells; the 25-year project produced dramatic patient improvements and was celebrated at a Los Angeles ceremony, while scientists warned about politicized attacks on science and other prize-winning work highlighted advances in gene therapies for blood disorders.

via The Guardian|

#breakthrough-prize #gene-therapy #leber-congenital-amaurosis