Search results for Otarmeni Gene Therapy

First Gene Therapy for Genetic Hearing Loss, Otarmeni, Gains FDA Approval

MONDAY, April 27, 2026 — In a historic leap for medical science, the U.S. Food and Drug Administration (FDA) has greenlit the first-ever gene therapy to treat a rare form of inheri...

EMA Accepts Otarmeni Gene Therapy for OTOF-Related Hearing Loss for Review

If approved, Regeneron's Otarmeni will be the first gene therapy for OTOF-related hearing loss in the European Union.

FDA approves Regeneron’s hearing loss gene therapy

Otarmeni, now cleared to treat a rare, inherited kind of hearing loss, is the first gene therapy cleared under the FDA’s “national priority” voucher program and will be offered to...

FDA Grants Accelerated Approval to Otarmeni Gene Therapy for OTOF-Related Hearing Loss

The adeno-associated virus vector-based gene therapy is indicated for pediatric and adult patients with severe-to-profound hearing loss due to biallelic OTOF gene variants.

Regeneron ushers in new genetic medicine era with groundbreaking gene therapy approval

Travis Smith was a very serious baby. Born completely unable to hear, his mother, Sierra, struggled to see his personality shine through his mute world.

Regeneron Gets Landmark FDA Approval for First Gene Therapy for Hearing Loss

Regeneron Pharmaceuticals’ Otarmeni is now approved for treating hearing loss from an ultra-rare genetic mutation found in an estimated 50 newborns per year. While Regeneron will o...

rAAV8 HMR-001 Boosts Liver Gene Therapy in Hemophilia

In a groundbreaking advancement for gene therapy, researchers have unveiled two novel bioengineered AAV8 vectors—HMR-001 and its codon-optimized counterpart HMR-001z—that demonstra...

FDA to reconsider shock rejection of cell therapy Ebvallo. Could uniQure be next?

After a surprise rejection in January, the FDA has agreed to reconsider a T-cell therapy based on the same single-arm trial that the agency had previously found problematic. The ca...

Long-Term SHIV Suppression Using AAV Gene Therapy

Leronlimab is an antiviral HIV drug that targets and blocks the CCR5 receptor, thus blocking HIV’s ability to invade immune cells. Most macaques that produced sufficient number of...

La FDA aprueba Otarmeni: La primera terapia génica para la sordera genética

La agencia reguladora estadounidense autorizó un fármaco que utiliza vectores virales para restaurar la proteína otoferlina en niños, permitiendo que el cerebro procese el sonido d...

Toloo Taghian Secures $3.2M Grant to Advance Gene Therapy for Rare Genetic Disorder

The National Institute of Neurological Disorders and Stroke (NINDS) has awarded Toloo Taghian, PhD, an assistant professor of genetic and cellular medicine as well as radiology at...

CAR T-cell therapy takes woman from bedridden to 'perfectly fine'

A woman with three different autoimmune conditions had all of them treated simultaneously by genetically modifying her immune cells to kill off the rogue ones causing problems

Cytokine‑Armored CAR T Cells Overcome Antigen Heterogeneity in Glioma Model

Scientists developed a cytokine-armored CAR T-cell therapy that helps the immune system better attack aggressive brain tumors in mice, which they paired with a CAR T strategy targe...

G-Link CAR-T Delivery Platform Showcased at ASGCT

The modular plug-and-play protein adapter developed in collaboration with Menachem Rubenstein, PhD, of the Weizmann Institute allows drug developers to cap and retarget existing le...

<![CDATA[FDA Approves Otarmeni Gene Therapy for OTOF-Related Hearing Loss]]>

Acute Myeloid Leukemia Therapy Improved by CRISPR Stem Cell Transplant

A Phase I/II clinical trial shows that a stem cell transplant that removes CD33 from donor cells using CRISPR can prevent cancer recurrence. The post Acute Myeloid Leukemia Therapy...

Breakthrough in CAR T Cell Therapy: Insights from Successfully Treated AML Patients

A groundbreaking advance in the treatment of acute myeloid leukemia (AML) heralds a new era in immunotherapy, addressing a long-standing challenge of targeting cancer cells without...

With $97M, Latus pursues a different kind of Huntington’s gene therapy

The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.

Gene Therapy Causes Patient to Grow Alarming Tumor

Tinkering with the genetic code of a living human is risky. The post Gene Therapy Causes Patient to Grow Alarming Tumor appeared first on Futurism.

Gene Therapy for Inherited Deafness Shows Lasting Hearing Restoration

A multicenter study found a one-time treatment targeting the OTOF gene restored hearing in most participants, with effects lasting up to 2.5 years.

Stem Cell Editing Programs the Immune System to Make Own Therapeutic Proteins

A new gene‑editing strategy programs hematopoietic stem cells to produce therapeutic antibodies and other proteins. The approach generates long‑lasting immunity in mice and could p...

Gene Therapy Proven to Shield Brain from TDP-43 Damage

An experimental gene therapy called SynCav1 can protect the central nervous system from the cognitive decline linked to TDP-43-related proteinopathy, a primary driver of FTD, ALS,...

Taysha to present preclinical data on Rett syndrome gene therapy

Radboudumc gives first-ever experimental T-cell therapy to pancreatic cancer patient

For the first time in the world, Radboudumc has treated a patient with pancreatic cancer using a new experimental immunotherapy that genetically modifies the patient’s own immune c...