Latest news with #OTOF
Yahoo
09-07-2025
- Health
- Yahoo
Researchers used gene therapy to restore hearing in adults
If you purchase an independently reviewed product or service through a link on our website, BGR may receive an affiliate commission. Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the subtlety of natural hearing. But researchers may have finally opened the door to an alternative option. According to new research, scientists have successfully used gene therapy to restore hearing in adults with congenital deafness, marking a major milestone in the quest to restore hearing in people of all ages. Today's Top Deals XGIMI Prime Day deals feature the new MoGo 4 and up to 42% off smart projectors Best deals: Tech, laptops, TVs, and more sales Best Ring Video Doorbell deals We know that gene therapy can be extremely powerful. Not only has gene editing opened the door for many potential treatments, but it has also paved the way for groundbreaking research like hypoallergenic cats, and more. This new treatment can restore hearing loss in adults by targeting a rare form of genetic hearing loss called OTOF-related deafness. This condition stems from mutations in the OTOF gene, which normally produces a protein called otoferlin. Otoferlin is critical for transmitting sound signals from the inner ear to the brain. That signal chain is broken in patients with this mutation. This leads to hearing loss from birth onward. Because the inner ear structures remain healthy, though, the condition is well suited for gene therapy treatments. To deliver a working copy of the OTOF gene, researchers used a harmless, modified virus that was released directly into the inner ear's sensory cells. This virus essentially acted as a courier, carrying the fixed gene into the cells so they can begin producing otoferlin and hopefully restore sound signaling. Initial tests in children showed promising results, but a bigger question remained: Could gene therapy work in older patients? To find out, the team launched a clinical trial involving 10 participants between the ages of 1 and 24, all diagnosed with OTOF-related deafness. The results were encouraging across the board—and especially noteworthy in older participants. Hearing improvements were both rapid and significant. On average, participants showed a 62% improvement in brainstem response tests and a 78% gain in behavioral hearing assessments. Some were able to detect the sounds of speech within just a few weeks. This research marks the first time gene therapy has been shown to restore hearing in both adolescents and adults. Side effects were reportedly mild and temporary, with no serious complications observed. Most commonly, patients experienced a brief drop in white blood cell counts. A full write-up on the research is available at The Conversation, as well as in the official published study available in Nature Medicine. More Top Deals Memorial Day security camera deals: Reolink's unbeatable sale has prices from $29.98 See the
Yahoo
06-07-2025
- Health
- Yahoo
Breakthrough gene therapy jab reverses hearing loss in weeks
A single jab of a breakthrough gene therapy could reverse hearing loss in people within weeks, according to new research. The cutting-edge therapy improved hearing in children and adults with congenital deafness or severe hearing impairment, with a 7-year-old regaining almost full hearing in a clinical trial, researchers from Sweden's Karolinska Institutet said. The clinical trial, detailed in the journal Nature Medicine, showed that a healthy copy of the OTOF gene injected in the inner ear improved hearing of all 10 participants. The small-scale trial included people who had a genetic form of deafness or severe hearing impairment caused by mutations in a gene called OTOF. These mutations cause a deficiency of the protein otoferlin, which plays a key role in transmitting sound signals from the ear to the brain. While the therapy seemed to work best in children, researchers said, it could benefit adults as well. In the trial, a synthetic, harmless version of the adeno-associated virus was used to deliver a properly functional OTOF gene to the inner ear via a single injection. The effects of the therapy were evident in the majority of patients, whose hearing recovered rapidly after just a month. After six months, researchers noted considerable hearing improvement in all participants, with their average volume of perceptible sound improving from 106 decibels to 52. Those between the ages of five and eight responded best to the treatment, the study found. One seven-year-old girl quickly recovered almost all her hearing, and she was able to hold daily conversations with her mother four months afterwards. 'This is the first time that the method has been tested in teenagers and adults,' Maoli Duan, an author of the study from Karolinska Institutet, said. "Hearing was greatly improved in many of the participants, which can have a profound effect on their life quality. We will now be following these patients to see how lasting the effect is.' Researchers also found that the treatment was safe and well-tolerated. Participants did not report any serious adverse reactions in the follow-up period of 6-12 months. The most common reaction was a reduction in the number of the immune system's neutrophils, a type of white blood cell. "OTOF is just the beginning," Dr Duan said, adding that researchers were working on other common genes behind deafness such as GJB2 and TMC1. 'These are more complicated to treat, but animal studies have so far returned promising results. We are confident that patients with different kinds of genetic deafness will one day be able to receive treatment.'
Yahoo
06-07-2025
- Health
- Yahoo
Breakthrough gene therapy jab reverses hearing loss in weeks
A single jab of a breakthrough gene therapy could reverse hearing loss in people within weeks, according to new research. The cutting-edge therapy improved hearing in children and adults with congenital deafness or severe hearing impairment, with a 7-year-old regaining almost full hearing in a clinical trial, researchers from Sweden's Karolinska Institutet said. The clinical trial, detailed in the journal Nature Medicine, showed that a healthy copy of the OTOF gene injected in the inner ear improved hearing of all 10 participants. The small-scale trial included people who had a genetic form of deafness or severe hearing impairment caused by mutations in a gene called OTOF. These mutations cause a deficiency of the protein otoferlin, which plays a key role in transmitting sound signals from the ear to the brain. While the therapy seemed to work best in children, researchers said, it could benefit adults as well. In the trial, a synthetic, harmless version of the adeno-associated virus was used to deliver a properly functional OTOF gene to the inner ear via a single injection. The effects of the therapy were evident in the majority of patients, whose hearing recovered rapidly after just a month. After six months, researchers noted considerable hearing improvement in all participants, with their average volume of perceptible sound improving from 106 decibels to 52. Those between the ages of five and eight responded best to the treatment, the study found. One seven-year-old girl quickly recovered almost all her hearing, and she was able to hold daily conversations with her mother four months afterwards. 'This is the first time that the method has been tested in teenagers and adults,' Maoli Duan, an author of the study from Karolinska Institutet, said. "Hearing was greatly improved in many of the participants, which can have a profound effect on their life quality. We will now be following these patients to see how lasting the effect is.' Researchers also found that the treatment was safe and well-tolerated. Participants did not report any serious adverse reactions in the follow-up period of 6-12 months. The most common reaction was a reduction in the number of the immune system's neutrophils, a type of white blood cell. "OTOF is just the beginning," Dr Duan said, adding that researchers were working on other common genes behind deafness such as GJB2 and TMC1. 'These are more complicated to treat, but animal studies have so far returned promising results. We are confident that patients with different kinds of genetic deafness will one day be able to receive treatment.'
The Independent
04-07-2025
- Health
- The Independent
Breakthrough gene therapy jab reverses hearing loss in weeks
A single jab of a breakthrough gene therapy could reverse hearing loss in people within weeks, according to new research. The cutting-edge therapy improved hearing in children and adults with congenital deafness or severe hearing impairment, with a 7-year-old regaining almost full hearing in a clinical trial, researchers from Sweden 's Karolinska Institutet said. The clinical trial, detailed in the journal Nature Medicine, showed that a healthy copy of the OTOF gene injected in the inner ear improved hearing of all 10 participants. The small-scale trial included people who had a genetic form of deafness or severe hearing impairment caused by mutations in a gene called OTOF. These mutations cause a deficiency of the protein otoferlin, which plays a key role in transmitting sound signals from the ear to the brain. While the therapy seemed to work best in children, researchers said, it could benefit adults as well. In the trial, a synthetic, harmless version of the adeno-associated virus was used to deliver a properly functional OTOF gene to the inner ear via a single injection. The effects of the therapy were evident in the majority of patients, whose hearing recovered rapidly after just a month. After six months, researchers noted considerable hearing improvement in all participants, with their average volume of perceptible sound improving from 106 decibels to 52. Those between the ages of five and eight responded best to the treatment, the study found. One seven-year-old girl quickly recovered almost all her hearing, and she was able to hold daily conversations with her mother four months afterwards. 'This is the first time that the method has been tested in teenagers and adults,' Maoli Duan, an author of the study from Karolinska Institutet, said. "Hearing was greatly improved in many of the participants, which can have a profound effect on their life quality. We will now be following these patients to see how lasting the effect is.' Researchers also found that the treatment was safe and well-tolerated. Participants did not report any serious adverse reactions in the follow-up period of 6-12 months. The most common reaction was a reduction in the number of the immune system's neutrophils, a type of white blood cell. "OTOF is just the beginning," Dr Duan said, adding that researchers were working on other common genes behind deafness such as GJB2 and TMC1. 'These are more complicated to treat, but animal studies have so far returned promising results. We are confident that patients with different kinds of genetic deafness will one day be able to receive treatment.'
Business Wire
01-07-2025
- Business
- Business Wire
Sensorion Announces Preliminary Positive Data from the First Cohort of the Audiogene Phase 1/2 Gene Therapy Clinical Trial
MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News: Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat and prevent hearing loss disorders, today announced preliminary positive data from the first cohort of the Phase 1/2 Audiogene clinical trial evaluating the low dose of SENS-501, the Company's gene therapy candidate being developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. The second cohort at a higher dose is ongoing and recruitment is close to being completed. The results from all patients dosed to date (5) confirm that SENS-501 and the corresponding surgical procedure are well tolerated by all participating infants and toddlers (aged 6 to 31 months and naive of cochlear implants at the time of the injection, as per study protocol) having received a gene therapy injection. Intracochlear administration of SENS-501 was uneventful, and no serious adverse events or serious side effects were reported. Three patients were enrolled into Cohort 1 and received a low dose of SENS-501 of 1.5 E 11 vg/vector/ear, corresponding to the minimally effective dose in preclinical studies. The primary objective is to assess the safety and feasibility of the intra-cochlear administration of SENS-501. In Cohort 1, early signs of hearing improvement were observed in Patient 3, aged 11 months at the time of injection. The clinical response observed in Patient 3 was evaluated using standard hearing tests carried out by the investigators (Auditory Brainstem Response ABR, Pure Tone Audiometry PTA, and Patient (Parents) Reported Outcomes PROs). Three-month data from the Patient 3 include: Positive ABR responses at two frequencies, with the best frequency reaching 70 dB. Improvement of hearing levels across two speech frequencies with best frequency reaching 90 dB level, per PTA. Meaningful changes in responses to sounds and voices as reported by the parents with an IT-MAIS score increase of 16 points (145% relative improvement from baseline), and met expected auditory milestones based on an age-based parent questionnaire and according to the patient's age (LittlEARS). The recruitment in Cohort 2, utilizing a second and higher dose level, is nearly complete. The Company plans to provide the next update when Cohort 2 data have reached sufficient maturity to determine next steps for the program. Professor Catherine Birman, ENT surgeon, otolaryngologist, and Senior Staff Specialist at the Children's Hospital at Westmead, Australia, commented: "I'm thrilled to report the preliminary Cohort 1 data of SENS-501 in the first infants and toddlers treated with this highly innovative therapy. Treatment with SENS-501 had a good safety profile and the onset of early auditory responses observed in Patient 3 of the first cohort is very encouraging, especially given the very low dose of vector injected, which is primarily intended at assessing the safety of the therapeutic and of the intracochlear surgical procedure. I look forward to Patient 3's next visit and continuing the Audiogene study with the second cohort to assess a higher dose of SENS-501. Treating children under 31 months of age and naive of cochlear implants is a much-needed undertaking, as restoring hearing in the first three years of childhood has the potential to result in de-novo language acquisition. We thank Sensorion for their commitment to this patient population.' The Phase 1/2 clinical trial Audiogene ( ID: NCT06370351), developed in the frame of the strategic partnership with the Institut Pasteur and led by Professor Natalie Loundon, Coordinating Investigator, M.D., Director of the Center for Research in Pediatric Audiology, Pediatric Otolaryngologist and Head and Neck Surgeon, Necker Enfants Malades, AP-HP, in Paris, France, aims to evaluate the safety and efficacy of an intra-cochlear injection of SENS-501 for the treatment of OTOF gene-induced hearing loss in paediatric patients aged 6 to 31 months and naive of cochlear implants at the time of the gene therapy treatment. Audiogene consists of a dose-escalation part, comprising two cohorts of three patients each, assessing a low dose of SENS-501 in Cohort 1 (1.5 E 11 vg/vector/ear) and a higher dose of SENS-501 in Cohort 2 (4.5 E 11 vg/vector/ear). The dose-escalation part will be followed by a dose-expansion cohort at the selected dose. While safety is the primary endpoint of the dose escalation study, Auditory Brainstem Response, twelve months following the injection, will be the primary endpoint for the dose expansion part. Audiogene is the first gene therapy clinical trial addressing a unique homogeneous population of infants and toddlers (aged 6 to 31 months and naive of cochlear implants at the time of the gene therapy injection). Addressing this young patient population aims at maximizing the chances of these infants and toddlers to acquire language (below three years old, when brain plasticity is optimal). Furthermore, and uniquely to Audiogene's gene therapy program, all enrolled patients should not have current or previous cochlear implantation in the treated or contralateral ear, allowing to best document the contribution of the gene therapy in speech development. About SENS-501 SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is defective, affected individuals are born with severe to profound hearing loss. The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to restore the normal process of converting sound into electrical signals, enabling patients to regain their hearing ability. Currently in the clinical research phase, this gene therapy program represents significant hope for families affected by this rare form of genetic deafness. SENS-501 (OTOF-GT) embodies a commitment to scientific innovation in the field of hearing, with the potential to dramatically improve the quality of life of patients suffering from genetic deafness. This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l'Audition. The project is partially financed by the French National Research Agency, through the 'investing for the future' program (ref: ANR-18-RHUS-0007). The OTOF gene targeted by the Audiogene trial was discovered in 1999 at the Institut Pasteur, by Prof. Christine Petit's team (Institut reConnect, Institut de l'Audition, Pasteur Institute), who also unraveled the pathophysiology of the corresponding deafness (DFNB9). About Sensorion Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. Sensorion's portfolio also comprises programs of a clinical-stage small molecule, SENS-401 (Arazasetron), for the treatment and prevention of hearing loss disorders. Sensorion's small molecule progresses in a Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) for the preservation of residual hearing. Sensorion, with partner Cochlear Limited, completed in 2024 a Phase 2a study of SENS-401 for the residual hearing preservation in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022. Disclaimer This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2024 full year report published on March 14, 2025, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.
