Congenital hearing loss

Sensorion Reports Full-Year 2023 Financial Results and Business Update

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Thursday, March 14, 2024
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Sensorion (Paris:ALSEN) (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, today reports full-year 2023 financial results and business update.

Key Points: 
  • Sensorion (Paris:ALSEN) (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, today reports full-year 2023 financial results and business update.
  • Nawal Ouzren, Chief Executive Officer of Sensorion, said: “Over the past months, Sensorion has successfully achieved all the major milestones on its roadmap.
  • In the first half of 2023, Sensorion achieved a major development milestone by completing the preclinical package, and successfully producing the GMP dual AAV SENS-501 clinical batches at 200L scale.
  • On April 6, 2023, Sensorion announced the candidate selection for GJB2-GT during its gene therapy focused R&D Day.

Sensorion Announces Approval to Initiate Lead Gene Therapy Candidate SENS-501 (OTOF-GT) into a Phase 1/2 Clinical Trial in some European Countries

Retrieved on: 
Friday, January 19, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Huber loss, Research, AAV, Hearing loss, EMA, Doctor of Philosophy, Deafness, Emeritus, Kavli Prize, Disorder, Physiology, Audiology, Safety, Hearing, Congenital hearing loss, Philosophy, CTA, Head, AP-HP, ABR, Pasteur Institute, Parent, Primate, Ageing, Cochlea, Patient, IHC, MD, European Medicines Agency, Partnership, Orphan, Language, Food, AP, FDA, Clinical trial, RHU, Pharmaceutical industry

Audiogene will also assess the clinical safety, performance, and usability of the administration device system under development in partnership with EVEON.

Key Points: 
  • Audiogene will also assess the clinical safety, performance, and usability of the administration device system under development in partnership with EVEON.
  • The design of the study will consist of two cohorts of 2 doses followed by an expansion cohort at the selected dose.
  • The CTA approval follows extensive preclinical studies assessing the safety and efficacy of SENS-501 and successful manufacturing of the gene therapy Drug Product for the clinical trial.
  • Sensorion presented preclinical data that indicated the potential for safe and efficient clinical translation of gene therapy for otoferlin delivered by a dual AAV vector.

Sensorion Announces Partnership With the Institut Pasteur to Accelerate Gene Therapy Programs Targeting Hearing Disorders Has Been Extended for Five Years

Retrieved on: 
Friday, January 5, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Kavli Prize, Partnership, Pasteur Institute, Research, Congenital hearing loss, Hearing loss, Trial of the century, Deafness, GJB2, Huber loss, Physiology, Emeritus, Patient, Diagnosis, Safety, Pharmaceutical industry

The agreement has been amended to be extended up to December 31, 2028, to promote additional development gene therapy programs.

Key Points: 
  • The agreement has been amended to be extended up to December 31, 2028, to promote additional development gene therapy programs.
  • SENS-501, the most advanced program within the partnership that targets deafness caused by mutations in the gene coding for otoferlin, defined as a priority in 2019, has met its objectives.
  • The successful completion of the efficacy preclinical package in the frame of the collaboration between Sensorion and the Institut Pasteur advanced the program with the development of the OTOF-GT product (SENS-501) towards clinical stage.
  • Three indications, all linked to GJB2 mutations, are currently being evaluated: early presbycusis, progressive hearing loss during childhood, and congenital hearing loss.

Regeneron Completes Acquisition of Decibel Therapeutics, Adding Promising Gene Therapy Programs for Hearing Loss

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Monday, September 25, 2023
STRC, Centerview Partners, CVR, Doctor of Philosophy, Hearing loss, Eastern Time Zone, Congenital hearing loss, European Medicines Agency, Gene editing, Clinical trial, GAAP, AAV, Wilmer Cutler Pickering Hale and Dorr, Wachtell, Lipton, Rosen & Katz, Gene silencing, Biologics license application, GJB2, Acquisition, Patient, CVR Partners, Computershare, Biology, Medicines and Healthcare products Regulatory Agency, Regulation of tobacco by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Regeneron Pharmaceuticals, Decibel

TARRYTOWN, N.Y., Sept. 25, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that it has successfully completed its acquisition of Decibel Therapeutics, Inc., strengthening the company’s gene therapy and auditory programs.

Key Points: 
  • TARRYTOWN, N.Y., Sept. 25, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that it has successfully completed its acquisition of Decibel Therapeutics, Inc., strengthening the company’s gene therapy and auditory programs.
  • The acquisition of Decibel builds on prior collaboration between the companies and includes three ongoing gene therapy programs targeting different forms of congenital, monogenic hearing loss.
  • Preclinical programs include AAV.103 for people with GJB2-related hearing loss and AAV.104 for people with stereocilin (STRC)-related hearing loss.
  • Following its acceptance of the tendered shares, Regeneron completed its acquisition of Decibel through a second step merger of Symphony Acquisition Sub, Inc., with and into Decibel.

Sensorion Reports 2023 First Half Results

Retrieved on: 
Wednesday, September 20, 2023
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In the first half of 2023, Sensorion achieved a major development milestone by completing the preclinical package, and successfully producing the GMP dual AAV OTOF-GT batches at 200L clinical scale.

Key Points: 
  • In the first half of 2023, Sensorion achieved a major development milestone by completing the preclinical package, and successfully producing the GMP dual AAV OTOF-GT batches at 200L clinical scale.
  • In April 2023, Sensorion announced the candidate selection for GJB2-GT during its R&D Day focusing on gene therapy.
  • The first patient was enrolled in December 2022 and Sensorion anticipates the publication of the preliminary results in H2 2023.
  • The annual accounts on June 30, 2023, drawn up according to IFRS standards and approved by the Board of Directors on September 18, 2023.

Decibel Therapeutics Reports Second Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Friday, August 11, 2023
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BOSTON, Aug. 11, 2023 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.

Key Points: 
  • In May 2023, Decibel announced the initiation of CHORD, a global Phase 1/2 dose escalation clinical trial of DB-OTO in pediatric patients.
  • Second Quarter 2023 Financial Results:
    Cash Position: As of June 30, 2023, cash, cash equivalents and available-for-sale securities were $78.3 million, compared to $104.6 million as of December 31, 2022.
  • Research and Development Expenses: Research and development expenses were $12.8 million for the second quarter of 2023, compared to $11.2 million for the same period in 2022.
  • General and Administrative Expenses: General and administrative expenses were $6.2 million for the second quarter of 2023, compared to $5.9 million for the same period in 2022.

Sensorion Submits Clinical Trial Application for OTOF-GT, its Lead Gene Therapy Candidate, in Europe

Retrieved on: 
Wednesday, July 19, 2023
Health, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, US Foods, Audiogenic, Hearing loss, Congenital hearing loss, European Medicines Agency, Cochlea, Clinical trial, Huber loss, CTA, AAV, IHC, EMA, CTAS, Patient, Hearing, Approved Drug Products with Therapeutic Equivalence Evaluations, Orphan, Ageing, FDA, Food, Safety, Pharmaceutical industry, Europe 1

The CTAs submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.

Key Points: 
  • The CTAs submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.
  • Sensorion’s OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss.
  • Géraldine Honnet, Chief Medical Officer of Sensorion, added: “We are excited to advance OTOF-GT, our first gene therapy program, to the clinic to address unmet medical need in the complex hearing space.
  • Hearing loss caused by mutations of the gene encoding for otoferlin is a debilitating disorder with currently no approved drugs.”

Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK

Retrieved on: 
Monday, July 10, 2023
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The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months.

Key Points: 
  • The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months.
  • The CTA submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.
  • Sensorion’s OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss.
  • Nawal Ouzren, Chief Executive Officer of Sensorion, commented: “This first gene therapy CTA filing is a major milestone for our OTOF-GT program and Sensorion’s broader gene therapy franchise.

Decibel Therapeutics Announces Approval of Clinical Trial Application by the Spanish Agency of Medicines and Medical Devices (AEMPS) to Initiate Clinical Development of Lead Gene Therapy Candidate DB-OTO

Retrieved on: 
Friday, May 12, 2023
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The Phase 1/2 dose escalation clinical trial, known as CHORD™, is designed to evaluate the safety, tolerability and efficacy of DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency. In addition to safety and tolerability endpoints, the auditory brainstem response (ABR) -- an objective, clinically accepted, physiologic measure of hearing sensitivity -- will be used as an efficacy endpoint in the clinical trial. The ABR, which was used to characterize dose-response of DB-OTO after intra-cochlear delivery in translational studies in animal models, provides an opportunity to rapidly assess hearing functionality and sensitivity.

Key Points: 
  • BOSTON, May 12, 2023 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced approval of its Clinical Trial Application (CTA) by the Spanish Agency of Medicines and Medical Devices (AEMPS) to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate.
  • Medicines and Healthcare Products Regulatory Agency (MHRA) in January 2023.
  • Decibel expects that clinical trial participants in the U.K. and Spain will be infants two years of age and younger.
  • In addition to safety and tolerability endpoints, the auditory brainstem response (ABR) -- an objective, clinically accepted, physiologic measure of hearing sensitivity -- will be used as an efficacy endpoint in the clinical trial.

Bionano Announces Publication that Demonstrates OGM’s Utility for Resolving Complex Genetic Variation in Inherited Retinal Disease

Retrieved on: 
Thursday, April 6, 2023
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Their results identified several pathogenic SVs had been overlooked in their initial genome analyses by NGS.

Key Points: 
  • Their results identified several pathogenic SVs had been overlooked in their initial genome analyses by NGS.
  • The publication illustrates the potential impact of combining NGS and OGM for more comprehensive variant analysis.
  • Genetic research in this area may help to improve the accuracy of diagnosis, prognostication, and treatment prospects of targeted therapeutics.
  • With new gene therapy and clinical trials related to IRD, research into the underlying genetic cause of IRDs is critical.