Audiogenic

Praxis Precision Medicines to Showcase Largest Pipeline of Precision Epilepsy Programs and Breadth of Commitment to Epilepsy Treatments at Upcoming Meetings

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화요일, 11월 28, 2023
Molecule, Clinical trial, Audiogenic, Conference, PAME, Drug discovery, EEG, SCN2A, Drug development, Cerebrum, DEE, AES, Mortality, American Epilepsy Society, SCN8A, Standard of care, Pharmacokinetics, Annual general meeting, CNS, Part, Biomarker, Family Gathering, Generalized tonic–clonic seizure, Central nervous system, Patient, Safety, BTS, ASM, MES, Pharmaceutical industry

Presentations at the American Epilepsy Society (AES) Annual Meeting, as well as related scientific and patient advocacy meetings, will cover new patient data, updates on clinical progress and new paradigms for developing epilepsy therapies.

Key Points: 
  • Presentations at the American Epilepsy Society (AES) Annual Meeting, as well as related scientific and patient advocacy meetings, will cover new patient data, updates on clinical progress and new paradigms for developing epilepsy therapies.
  • ET [Platform D | Epilepsy Therapies]
    Summary: Combined preclinical and clinical data demonstrate PRAX-628 is differentiated from standard of care, with the potential to be best-in-class for focal epilepsy.
  • Summary: This study provides novel insights into the clinical validity of commonly used preclinical seizure models across the clinical epilepsy spectrum.
  • Oral presentation highlighting clinical updates from our PRAX-562 small molecule platform at the annual clinician, researcher, and family gathering for SCN8A

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

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수요일, 7월 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: 
월요일, 7월 10, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, US Foods, Audiogenic, Dental implant, Congenital hearing loss, Health care, European Medicines Agency, Cochlea, Clinical trial, Quality of life, Mutation, CTA, AAV, IHC, EMA, Patient, Hearing, Approved Drug Products with Therapeutic Equivalence Evaluations, Medicines and Healthcare products Regulatory Agency, Orphan, Ageing, FDA, Safety, Food, MHRA, Pharmaceutical industry, Europe 1

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.