AAV

SpliceBio Appoints David Favre, D.V.M., Ph.D., as Chief Development Officer to Accelerate Development of its Lead AAV Gene Therapy Program

Retrieved on: 
Tuesday, June 20, 2023
Protein splicing, University, GSK plc, GSK, Drug development, Doctor of Philosophy, HIV, Stargardt disease, Education minister, Stargardt, Immunotherapy, St. Andrews University (North Carolina), AAV, CDO, Biology, Translational medicine, DSM-IV codes, UCSF, IND, Gladstone Institutes, Gene, Bangladesh Technical Education Board, Medical device, Pharmaceutical industry, Vaccine, Science, Microbiology

Dr. Favre brings more than 25 years of experience in gene therapy, immunology and drug development throughout academia and the biopharma industry.

Key Points: 
  • Dr. Favre brings more than 25 years of experience in gene therapy, immunology and drug development throughout academia and the biopharma industry.
  • He joins SpliceBio from Innoskel, where he served as Chief Scientific Officer.
  • David will focus on advancing the company's lead AAV gene therapy program for Stargardt disease towards clinical development and expanding the company's Protein Splicing platform and pipeline to other genes that exceed the packaging capacity of AAV vectors."
  • David Favre, CDO of SpliceBio, commented: "SpliceBio has built an impressive gene therapy platform that addresses one of the most fundamental challenges facing the gene therapy field today: the limited packaging capacity of AAV vectors.

Evox Therapeutics Acquires Exosome AAV Technology and Intellectual Property

Retrieved on: 
Tuesday, June 20, 2023
Vivisection, Engineering, Exosome, Acquisition, Therapy, Cell, AAV, Vaccine, Pharmaceutical industry

OXFORD, United Kingdom, June 20, 2023 (GLOBE NEWSWIRE) -- Evox Therapeutics Ltd (‘Evox’ or the ‘Company’), a leading exosome therapeutics company, has purchased Codiak Biosciences engEx-AAV™ technology platform, including all intellectual property rights therein, as well as secured exclusive access to certain engineering and manufacturing rights associated with it. This technology enables active loading of adeno-associated virus (AAV) into exosomes followed by their release when the AAV-loaded exosomes are delivered into recipient cells. This has been demonstrated to be an effective way to improve AAV delivery, to increase the breadth of expression in pre-clinical models, and to shield AAVs from neutralizing antibodies.

Key Points: 
  • OXFORD, United Kingdom, June 20, 2023 (GLOBE NEWSWIRE) -- Evox Therapeutics Ltd (‘Evox’ or the ‘Company’), a leading exosome therapeutics company, has purchased Codiak Biosciences engEx-AAV™ technology platform, including all intellectual property rights therein, as well as secured exclusive access to certain engineering and manufacturing rights associated with it.
  • This technology enables active loading of adeno-associated virus (AAV) into exosomes followed by their release when the AAV-loaded exosomes are delivered into recipient cells.
  • “We are pleased to now own this technology and look forward to applying it in future exosome AAV programs.
  • This acquisition further adds to our dominant intellectual property portfolio of patents that cover key aspects needed to develop and commercialise exosome therapeutics including their design, composition, and manufacture,” said Per Lundin, Ph.D., Chief Business Officer & Co-Founder of Evox Therapeutics.

CureDuchenne, Muscular Dystrophy Association, and Parent Project Muscular Dystrophy Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, June 15, 2023
Muscular Dystrophy Association, University, Pediatrics, Duchenne muscular dystrophy, Doctor of Philosophy, Muscular dystrophy, Sarepta, Myasthenia gravis, Antibody, AAV, Associate, Trial of the century, Organization, DMD, Patient, PPMD, Immunoglobulin G, Alfa Romeo, MDA, FDA, Safety, Disease, Vaccine, Pharmaceutical industry, Efgartigimod alfa, Duchenne, Research, Pfizer

Positive results from this study may be applicable to the translation of AAV gene therapies for other neuromuscular diseases.

Key Points: 
  • Positive results from this study may be applicable to the translation of AAV gene therapies for other neuromuscular diseases.
  • "We are just beginning to realize the impact of gene therapies that can treat the underlying root cause of genetic diseases.
  • Several companies including Sarepta, Solid Biosciences, Pfizer, and Regenxbio have undertaken clinical trials with truncated dystrophin constructs for gene therapy.
  • “Gene therapy delivered by AAV represents a potentially transformative therapy for patients, but some individuals are barred from accessing these AAV delivered therapies due to pre-existing antibodies.

Taysha Gene Therapies to Host Virtual R&D Day on Lead Clinical Investigational Programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome

Retrieved on: 
Thursday, June 15, 2023
Central nervous system, Natural history, Rett syndrome, AAV, CNS, Assistant, University of Texas Southwestern Medical Center, GAN, Biology, Neurology, Safety, Pharmaceutical industry, DALLAS, MD

DALLAS, June 15, 2023 (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 it will host a virtual R&D Day on Wednesday, June 28, 2023 at 10:00 AM ET to discuss updates on TSHA-120, a self-complimentary intrathecally delivered investigational AAV9 gene therapy in clinical evaluation for GAN, and TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome.

Key Points: 
  • In addition, Taysha leadership will provide a clinical update on the investigational TSHA-102 program, including the initial safety observations of TSHA-102 from the first patient recently dosed in the Phase 1/2 REVEAL trial .
  • The REVEAL trial is evaluating the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome.
  • More detailed clinical updates on the first patient will be provided in the third quarter of this year following the initial review of available safety data by the Independent Data Monitoring Committee.
  • A live question and answer session will follow the formal presentations.

LEXEO Therapeutics Announces Completion of First Cohort and Dosing in Second Cohort in SUNRISE-FA, a Phase 1/2 Clinical Trial of LX2006 for the Treatment of Friedreich’s Ataxia Cardiomyopathy

Retrieved on: 
Tuesday, June 13, 2023
Ataxia, Serum, Biomarker, AAV, Data monitoring committee, Patient, Toxic leukoencephalopathy, FA, Therapy, Death, Caregiver, Safety, Vaccine, Pharmaceutical industry, Hospital

NEW YORK, June 13, 2023 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease, announced today the completion of the first dose cohort and the dosing of the first patient in the second dose cohort in SUNRISE-FA, a Phase 1/2 clinical trial of LX2006 in patients with Friedreich’s ataxia (FA) cardiomyopathy.

Key Points: 
  • In the first dose cohort, LX2006 has been well tolerated with no unexpected events or toxicities observed.
  • Following the Data Safety Monitoring Board recommendation to proceed, investigators have initiated dosing in the second cohort.
  • “New treatment approaches, like LEXEO’s LX2006 gene therapy candidate, are critical for individuals and caregivers confronted with the debilitating realities of FA,” said Friedreich’s Ataxia Research Alliance Chief Executive Officer Jennifer Farmer.
  • For additional information about the trial, visit www.clinicaltrials.gov using the study identifier NCT05445323 .

Charles River Laboratories Launches Off-the-Shelf Lentiviral Vector Packaging Plasmids

Retrieved on: 
Tuesday, June 13, 2023
Research, Medical Devices, Genetics, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, Science, GMP, Associate Director of National Intelligence and Chief Information Officer, DNA, LVV, Plasmid, AAV, ATMP, CRL, NYSE, Tecmar, Vaccine

Charles River Laboratories International, Inc. (NYSE: CRL) today announced the launch of its off-the-shelf lentiviral vector (LVV) packaging plasmids, providing a streamlined solution to the challenges associated with plasmid sourcing in cell and gene therapies.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) today announced the launch of its off-the-shelf lentiviral vector (LVV) packaging plasmids, providing a streamlined solution to the challenges associated with plasmid sourcing in cell and gene therapies.
  • Charles River’s off-the-shelf packaging plasmids offer an alternative solution to these hurdles with immediate availability, reduced development costs and simplified supply chains that streamline and secure lentiviral vector production.
  • In addition to its off-the-shelf offerings, Charles River provides services to meet customer specific needs such as regulatory requirements or product characteristics.
  • Combined with the Company’s legacy testing services, Charles River offers an industry-leading “concept-to-cure” solution for advanced therapies.

NIH Approves Second Phase of Funding to Support IND-enabling Work for Ambulero Gene Therapy to Treat Vascular Disease

Retrieved on: 
Monday, June 12, 2023
Health, Genetics, Clinical Trials, Research, Science, Cardiology, Biotechnology, University, NIH, Amputation, Catalysis, University of Florida, National Heart, Lung, and Blood Institute, Vasculitis, IP, Lung, AAV, Doctor of Philosophy, Bangladesh Technical Education Board, Peripheral artery disease, NHLBI, US, Partner, MD, FACS, IND, Pharmaceutical industry

Ambulero, Inc. is developing a first-in-class gene and cell therapy platform for treating serious vascular diseases and non-healing wounds where amputation is a care option.

Key Points: 
  • Ambulero, Inc. is developing a first-in-class gene and cell therapy platform for treating serious vascular diseases and non-healing wounds where amputation is a care option.
  • Drs.
  • Velazquez and Liu are focused on advancing E-selectin/AAV gene therapies for the treatment of arterial occlusive diseases such as Peripheral Artery Disease with Chronic Limb Threatening Ischemia.
  • For more information about Ambulero, please see www.ambulero.com
    View source version on businesswire.com: https://www.businesswire.com/news/home/20230612819499/en/

Genezen Announces the Appointment of Key Executive Leaders, Expanding Viral Vector Capabilities

Retrieved on: 
Tuesday, June 13, 2023
LV, CMC, DRS, Thermo Fisher Scientific, Fujifilm Diosynth Biotechnologies, AAV, CCO, CDMO, CGMP, Knowledge, Growth, Therapy, Management, Vaccine, RV

Dr. D'Costa and Dr. O'Brien both bring remarkable expertise in gene and cell therapy manufacturing to supplement Genezen's already experienced leadership team.

Key Points: 
  • Dr. D'Costa and Dr. O'Brien both bring remarkable expertise in gene and cell therapy manufacturing to supplement Genezen's already experienced leadership team.
  • Steve Favaloro, President and CEO of Genezen, said, "Adding world renowned gene and cell therapy manufacturing experts like Drs.
  • Genezen is well positioned to develop new AAV vector production capabilities to supplement its deep expertise in LV and RV vectors."
  • Genezen has a best-in-class technical and operational team that is ready to serve clients' complex manufacturing needs."

Beacon Therapeutics launches with £96 million ($120 million) to develop a new generation of gene therapies for retinal diseases resulting in blindness

Retrieved on: 
Monday, June 12, 2023
BRC, US Foods, Vance DeGeneres, University, AMD, Food, Paratriathlon, Clinical trial, Retinitis pigmentosa, CRD, Retina, GTPase, AAV, IVT, Intravitreal administration, Oxford Sciences Enterprises, Cadherin related family member 3, Nightstar, Protein, Professor, Acquisition, Patient, McLaren, Partnership, VISTA, Knowledge, Entrepreneurship, RPGR, Therapy, FDA, OSE, University of Oxford, Muscular dystrophy, Vaccine, Pharmaceutical industry, Ophthalmology, Beacon

XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.

Key Points: 
  • XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
  • Beacon Therapeutics’s first pre-clinical asset is an intravitreally (IVT) delivered novel AAV based program for dry Age-related Macular Degeneration (dry AMD).
  • Beacon Therapeutics will be led by David Fellows, the former Chief Executive of Nightstar Therapeutics with over 40 years’ experience in the ophthalmology field.
  • Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data.

Candel Therapeutics Appoints Experienced Manufacturing Leader Nicoletta Loggia, PhD, RPh to its Board of Directors

Retrieved on: 
Thursday, June 8, 2023
Drug development, University, Gene, CMC, Survival, Quality of life, AAV, Pharmacist, Lentivirus, Novartis, MD, Patient, Cell, University of Pavia, Therapy, CADL, Prostate cancer, Orchard, Candel, Doctor of Philosophy, Fellow of the Academy of Medical Sciences

“We are pleased to welcome Nicoletta as a new member of Candel’s Board,” said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer (CEO) of Candel.

Key Points: 
  • “We are pleased to welcome Nicoletta as a new member of Candel’s Board,” said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer (CEO) of Candel.
  • “Her wealth of strategic and hands-on industry experience in drug development and manufacturing makes her an important addition to our Board as we advance our late-stage oncology programs.
  • She oversaw the integration of the CAR-T teams and AveXis’ AAV technical capabilities into Novartis’ technical research and development.
  • She is also a registered pharmacist and member of the UK Royal and Italian Pharmaceutical societies.