AAV

Adverum Biotechnologies Appoints Romuald Corbau, Ph.D. as Chief Scientific Officer

Retrieved on: 
Monday, January 29, 2024
Drug, Conditional sentence, Spark Therapeutics, Translation, Patient, Therapy, Employment, Paratriathlon, AAV, Universities of Lille, Innovation, Pfizer, LUNA, AMD, Adverum Biotechnologies, Drug discovery, CNS, Pharmaceutical industry

REDWOOD CITY, Calif., Jan. 29, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced that Romuald “Romu” Corbau, Ph.D., has joined Adverum as the company’s chief scientific officer.

Key Points: 
  • REDWOOD CITY, Calif., Jan. 29, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced that Romuald “Romu” Corbau, Ph.D., has joined Adverum as the company’s chief scientific officer.
  • “On behalf of the entire team at Adverum, I am delighted to welcome Dr. Corbau as the newest member of our executive leadership team.
  • Dr. Corbau joins Adverum from GenEdit, where he served as Chief Scientific Officer.
  • Prior to joining GenEdit, Dr. Corbau progressed from Vice President of Research to Chief Scientific Officer at Freeline, a clinical stage company developing AAV-vector mediated systemic gene therapies for chronic diseases.

NAYA Biosciences To Acquire Clinical Stage Gene Therapy Program for Leber's Hereditary Optic Neuropathy (LHON)

Retrieved on: 
Tuesday, January 23, 2024
Patient, Regenerative medicine, Fertility, Paratriathlon, AAV, Intercept Pharmaceuticals, Kite Pharma, University, Mitochondrion, Term sheet, LHON, Shearson, Acquisition, Safety, Vice, Intellectual property, FDA, Capsid, Letter, Arsenic trioxide, Paramount, Pharmaceutical industry

“The NAYA leadership team brings an agile entrepreneurial platform, broad development and commercialization experience, and access to public capital, which will unlock the potential of our promising AAV gene therapy platform for mitochondrial genetic diseases.

Key Points: 
  • “The NAYA leadership team brings an agile entrepreneurial platform, broad development and commercialization experience, and access to public capital, which will unlock the potential of our promising AAV gene therapy platform for mitochondrial genetic diseases.
  • The program has received over $6 million in grant funding to date and qualifies for Regenerative Medicine Advanced Therapy (RMAT) designation and multiple priority FDA review vouchers.
  • The combination of multiple orphan indications, multiple routes of administration, and multiple AAV serotypes applicable supports a broad gene therapy platform for mitochondrial orphan diseases.
  • The Florida Biotechnologies Transaction is contingent on the closing of the INVO Merger and sufficient financing to further develop the gene therapy programs from Florida Biotechnologies.

Taysha Gene Therapies Announces Poster Presentation on TSHA-102 in Rett Syndrome at Upcoming British Paediatric Neurology Association 2024 Annual Conference

Retrieved on: 
Monday, January 22, 2024
Patient, AAV, Rett syndrome, Conference, Central nervous system, Adolescence, CNS

DALLAS, Jan. 22, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that it will present clinical data on its TSHA-102 program in evaluation for Rett syndrome during an oral poster presentation at the upcoming British Paediatric Neurology Association (BPNA) 2024 Annual Conference, taking place in Bristol, England from January 24-26, 2024.

Key Points: 
  • DALLAS, Jan. 22, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that it will present clinical data on its TSHA-102 program in evaluation for Rett syndrome during an oral poster presentation at the upcoming British Paediatric Neurology Association (BPNA) 2024 Annual Conference, taking place in Bristol, England from January 24-26, 2024.
  • The presentation will include previously disclosed clinical data from the first two adult patients with stage four Rett syndrome dosed with TSHA-102 in the REVEAL Phase 1/2 adolescent and adult trial in Canada.
  • The Company expects to provide further updates on available clinical data from cohort one (low dose) in the REVEAL adolescent and adult trial in the first quarter of 2024.
  • Oral poster presentation details are as follows:

Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote

Retrieved on: 
Monday, January 29, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Mortality, Death, HSCT, Patient, Legislation, GALC, Advisory Committee, AAV, Psychosine, ESGCT, Data, Gene, DSM-IV codes, Neurodegenerative disease, Disease, Safety, Central nervous system, Sphingolipid, Physician, Krabbe disease, Cell, Human services, Parent, Nervous system, Clinical trial, Forge, Peripheral nervous system, Hematopoietic stem cell transplantation, Brain, UCBT, Pharmaceutical industry

Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.

Key Points: 
  • Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.
  • Previously published data have demonstrated that patients with Krabbe treated with HSCT demonstrate increased lifespan and stabilization of neurodegenerative disease in the central nervous system.
  • FBX-101, an investigational adeno-associated viral (AAV) gene therapy, has been designed to address the peripheral nerve disease not corrected by HSCT.
  • As a result, after a year of patient and foundation advocacy, Krabbe disease is again being voted on for potential inclusion on the RUSP.

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.

Advanced Medicine Partners Launches to Set New Standard for Manufacturing and Testing Advanced Medicines

Retrieved on: 
Wednesday, January 17, 2024
Health, Genetics, Other Health, General Health, Research, Science, Pharmaceutical, CMC, Patient, Amgen, GLP, Biotechnology, AAV, Novartis, GMP, Medicine, IND, Therapy, ARCH Venture Partners, Novartis Gene Therapies, Pharmaceutical industry

“Advanced Medicine Partners is a science- and mission-driven organization made up of one of the most experienced genetic medicines teams in the industry, and we are driven to deliver products and services that aim to set a new standard in the manufacturing and testing of innovative therapies,” said Andrew Knudten, CEO of Advanced Medicine Partners.

Key Points: 
  • “Advanced Medicine Partners is a science- and mission-driven organization made up of one of the most experienced genetic medicines teams in the industry, and we are driven to deliver products and services that aim to set a new standard in the manufacturing and testing of innovative therapies,” said Andrew Knudten, CEO of Advanced Medicine Partners.
  • Advanced Medicine Partners’ process has been used with multiple genes of interest as well as multiple wild-type AAV serotypes and engineered capsids.
  • Advanced Medicine Partners brings extensive operational experience from Jaguar Gene Therapy and other biotechnology companies, including AveXis, Novartis and Amgen.
  • In addition to Advanced Medicine Partners’ manufacturing and analytical testing capabilities, the company offers GMP tech transfer capabilities and virtual CMC support.

SCIENTISTS AT THE WCBP CONFERENCE HIGHLIGHT ADVANCEMENTS IN PROTEIN BIOTHERAPEUTIC CHARGE HETEROGENEITY CHARACTERIZATION USING THE MauriceFlex™ SYSTEM

Retrieved on: 
Wednesday, January 24, 2024
Patient, Mass spectrometry, Capsid, AAV, Sodium dodecyl sulfate, Spacecraft, Acceleration, Enzyme, Therapy, IEX, Fine chemical

The focus of these presentations was on the transformative impact of Bio-Techne's MauriceFlex™ system on their biotherapeutic development processes.

Key Points: 
  • The focus of these presentations was on the transformative impact of Bio-Techne's MauriceFlex™ system on their biotherapeutic development processes.
  • MauriceFlex™, known for its automated capillary isoelectric focusing (icIEF), also enables protein charge fractionation, allowing charge variant characterization in downstream analytics like mass spectrometry.
  • Like the Maurice™ system, the MauriceFlex™ also allows protein size analysis with capillary electrophoresis sodium dodecyl sulfate (CE-SDS).
  • "The discussions at the conference, particularly regarding the MauriceFlex™ system, reflect significant progress in the advancements of analytical techniques used in biotherapeutic development," said Will Geist, President of Bio-Techne's Protein Sciences Segment.

Viral Vector Manufacturing Success Stories: Onboarding to GMP Production, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Thursday, January 18, 2024
Doctor of Philosophy, Oxford Biomedica, Q&A, Risk, AAV, Quality control, Process, Design of experiments, GMP, Vaccine

TORONTO, Jan. 18, 2024 /PRNewswire-PRWeb/ -- Join this webinar to learn how to mitigate risk in viral vector manufacturing with tailored solutions and a flexible approach. Discover how unique and transferable technical capabilities in multiple viral vector types can significantly accelerate a development path and future-proof gene therapy products.

Key Points: 
  • In this free webinar, learn about client success stories from onboarding to GMP manufacturing of viral vectors for clinical use.
  • TORONTO, Jan. 18, 2024 /PRNewswire-PRWeb/ -- Join this webinar to learn how to mitigate risk in viral vector manufacturing with tailored solutions and a flexible approach.
  • They will share success stories of optimized production processes and successful transfer into good manufacturing practice (GMP).
  • Join this webinar to learn about successful case studies in viral vector manufacturing that use tailored solutions and a flexible approach.

Charles River Launches Rep/Cap Plasmids to Streamline Adeno-Associated Viral Vector Manufacturing

Retrieved on: 
Tuesday, January 16, 2024
Health, Medical Devices, Infectious Diseases, Genetics, General Health, Pharmaceutical, Biotechnology, Miami Beach Convention Center, Senior, CRL, CMC, Regulation, Patient, CTA, LVV, DNA, AAV, Education minister, GMP, Gene, Tecmar, IND, COA, Doctor of Philosophy, Investigational New Drug, RC2, Cell, CDMO, Commercialization, Plasmid, Pharmaceutical industry

Charles River Laboratories International, Inc. (NYSE: CRL) today announced the launch of its off-the-shelf Rep/Cap plasmid offering, designed to streamline adeno-associated virus (AAV)-based gene therapy programs.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) today announced the launch of its off-the-shelf Rep/Cap plasmid offering, designed to streamline adeno-associated virus (AAV)-based gene therapy programs.
  • Charles River will officially launch the off-the-shelf Rep/Cap plasmid offering during Advanced Therapies Week , January 16-19, at the Miami Beach Convention Center.
  • Join Dr. Baghirzade in the Gene Therapy Manufacturing Track as part of the Regulatory Alignment for Gene Therapy Manufacturing session to learn how to:
    Ensure alignment with phase-appropriate plasmid DNA, AAV, and lentiviral vector (LVV) manufacturing best practices
    Matthew Hewitt, B.A.
  • Charles River has significantly broadened its cell and gene therapy portfolio with several acquisitions and capacity expansions to simplify complex supply chains and meet the growing global demand for plasmid DNA, viral vector, and cell therapy services.

Taysha Gene Therapies Announces First Pediatric Patient Dosed with TSHA-102 in REVEAL Phase 1/2 Pediatric Trial in Rett Syndrome

Retrieved on: 
Wednesday, January 10, 2024
Ageing, Patient, CTA, MHRA, Medication, Principal, MECP2, Health care, Rush University Medical Center, AAV, Female, Rett syndrome, European Commission, Orphan drug, Risk, Mutation, Disease, Safety, Central nervous system, Caregiver, Fast Track, FDA, Index, Neurology, Adolescence, MTD, CNS, Pharmaceutical industry

DALLAS, Jan. 10, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that the first pediatric patient has been dosed with TSHA-102 in the REVEAL Phase 1/2 pediatric trial in the United States (U.S.) evaluating the safety and preliminary efficacy of TSHA-102 in stage three female patients 5-8 years of age with Rett syndrome. The Company also announced the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has authorized the Clinical Trial Application (CTA) for TSHA-102 in pediatric patients, enabling expansion of the ongoing U.S. REVEAL pediatric trial into the U.K.

Key Points: 
  • Initiation of REVEAL pediatric trial in the U.S. broadens the clinical evaluation of TSHA-102 to female patients 5-8 years old with stage three Rett syndrome
    MHRA authorized the CTA for TSHA-102 in pediatric patients with Rett syndrome, enabling expansion of ongoing U.S.
  • “Dosing the first pediatric patient with Rett syndrome marks an important step forward in our efforts to broaden the clinical evaluation of TSHA-102 to younger patients with earlier stages of Rett syndrome.
  • “The pediatric trial will build on our ongoing REVEAL adolescent and adult trial, where early data demonstrated improvements across multiple clinical domains in adult patients with the most advanced stage of disease.
  • TSHA-102 is also being evaluated in the ongoing first-in-human REVEAL Phase 1/2 adolescent and adult trial in females aged 12 and older with Rett syndrome in Canada.