A British toddler has had her hearing restored after becoming the first person in the world to take part in a pioneering gene therapy trial with Regeneron, in a development that doctors say marks a new era in treating deafness. An incredible breakthrough from Regeneron and the original Decibel Therapeutics team, whom Regeneron acquired in August 2023. Opal Sandy was born unable to hear anything due to auditory neuropathy, a condition that disrupts nerve impulses travelling from the inner ear to the brain and can be caused by a faulty gene. After receiving an infusion containing a working copy of the gene during groundbreaking surgery that took just 16 minutes, the 18-month-old can hear almost perfectly and enjoys playing with toy drums. Watch a heart warming video from Sky News with Opal and her parents. #regeneron #decibeltherapeutics #healthimpact #genetherapy https://lnkd.in/edyQq3ns
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🔹In a major medical breakthrough, a British baby born with a rare genetic condition that left her completely deaf has regained her ability to hear thanks to an innovative gene therapy. Performed at Addenbrooke's Hospital in Cambridge, the therapy involved administering a harmless virus that carries genetic material necessary for auditory functioning directly into the cochlea. This treatment marks a historic moment, with Opal being the youngest person and the first person in the world to benefit from this technique, opening up new possibilities for the treatment of deafness without relying on external devices. Find out more about this important medical breakthrough here 👉🏼 https://lnkd.in/dbnvMxcQ #techtitute #therapeutics #medicine #deafness
In a world first, 16-min gene therapy restores British child's hearing
interestingengineering.com
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‘Autosomal recessive deafness 9, caused by mutations of the OTOF gene, is characterised by congenital or prelingual, severe-to-complete, bilateral hearing loss. However, no pharmacological treatment is currently available for congenital deafness. In this Article, we report the safety and efficacy of gene therapy with an adeno-associated virus (AAV) serotype 1 carrying a human OTOF transgene (AAV1-hOTOF) as a treatment for children with autosomal recessive deafness 9.’ https://lnkd.in/d4EY99gV
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
thelancet.com
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For those who are interested in gene therapy, cell therapy, retinal diseases or ophthalmology in general
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This is a most important milestone in the development of EG427
We have received Investigational New Drug (IND) clearance from the U.S. FDA for our gene therapy EG110A! This first-in-human phase 1b/2a clinical study with EG110A is starting for the treatment of Neurogenic Detrusor Overactivity. This makes EG 427 the second company with active clinical trial with non-replicative HSV vectors, and the leader in neurology. “This IND clearance for our first product is a major milestone for EG 427, opening the way for clinical development of EG110A across a series of medically important but neglected neuro-urology pathologies,” says Philippe Chambon MD, PhD, CEO at EG 427 . Click below for the full press release. #biotech #genetherapy #drugdevelopment #bladderhealth
First gene therapy for prevalent pathologies in neurology receives FDA IND clearance - EG 427 initiating first-in-human clinical study
eg427.com
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Check out this new publication from Drs. Akhter, Bankiewicz, and Lonser: "Real-Time Magnetic Resonance Imaging During Convective Gene Therapy Perfusion of the Brain." Here they describe the critical importance of real-time imaging of convective perfusion of the brain for gene therapy in JAMA Surgery for the treatment of neurological disorders. https://go.osu.edu/CjKa
Real-Time MRI During Convective Gene Therapy Perfusion of the Brain
jamanetwork.com
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Gene therapy restoration of hearing in deaf children - how it works & where it's headed...
A cure for deafness?
cell.com
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AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial Autosomal recessive deafness 9, caused by mutations of the OTOF gene, is characterised by congenital or prelingual, severe-to-complete, bilateral hearing loss. However, no pharmacological treatment is currently available for congenital deafness. In this Article, we report the safety and efficacy of gene therapy with an adeno-associated virus (AAV) serotype 1 carrying a human OTOF transgene (AAV1-hOTOF) as a treatment for children with autosomal recessive deafness 9. Findings Between Oct 19, 2022, and June 9, 2023, we screened 425 participants for eligibility and enrolled six children for AAV1-hOTOF gene therapy (one received a dose of 9 × 1011 vector genomes [vg] and five received 1·5 × 1012 vg). All participants completed follow-up visits up to week 26. No dose-limiting toxicity or serious adverse events occurred. In total, 48 adverse events were observed; 46 (96%) were grade 1–2 and two (4%) were grade 3 (decreased neutrophil count in one participant). Five children had hearing recovery, shown by a 40–57 dB reduction in the average auditory brainstem response (ABR) thresholds at 0·5–4·0 kHz. https://lnkd.in/eX3d4fZw
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
thelancet.com
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On this week's podcast episodes, I'm joined by Dr. Khan, M.D., a cell and gene therapy specialist with a visionary approach that is changing the way we perceive healthcare worldwide. Khan has cemented his reputation as a regenerative medicine expert and a driven entrepreneur. In this episode, we discuss: ✔ What is stem cell therapy? ✔ Why the standard treatment for orthopedic injuries is wrong. ✔ How to heal faster with cutting-edge medicine. ✔ Can you use gene therapy to reverse aging? Tune in today through the link in the comments!
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Chief Executive Officer & CSO, LumaCyte | Innovative Scientist and Entrepreneur | Analytical Expert | Biotechnology Professional
Norrie disease is a rare but devastating genetic disorder that causes blindness and hearing loss, and it primarily affects boys. They are born blind and from about 12 years of age develop progressive hearing loss. While cochlear implants can help, they are only partially effective against norrie disease, leaving may young people without viable solutions. However, gene therapy has the potential to correct this: a recent mouse-based study found that it can prevent the death of the vitally important hair cells in the cochlea, preventing the hearing loss associated with the disease. This could offer a valuable alternative for patients, allowing them to live fuller lives. #NorrieDisease #GeneTherapy
Gene Therapy Developed for Hearing Loss Caused by Rare Disease
technologynetworks.com
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Sound On! Major news for the ~1 in 1,000 children born with genetic hearing loss👂 Dr. Chen's team has unlocked hearing for those with congenital deafness by targeting the OTOF gene mutation. Their pioneering therapy corrects the mutated gene that causes deafness. By splitting the large gene and delivering it via vectors into cells, they enabled sound transmission to the brain. This isn't just science; it's hope, sculpted into reality, offering a future where hearing loss is no longer a lifelong sentence. Watch the video here 🔊 : https://lnkd.in/gKeMAiBU #hearingloss #genetherapy #medicalresearch #biotech #healthcare #deafness
Thrilled to co-lead an endeavor with Dr. Yilai Shu of Shanghai Fudan Eye & ENT Hospital to start the first OTOF gene therapy clinical trial with the restoration of hearing in children who were completely deaf before the treatment, with a publication in The Lancet (https://lnkd.in/dpGwYV6w). We are entering a new era of novel treatments for genetic hearing loss.
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
thelancet.com
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