Gene therapy

Excellos Announces Acceleration of Commercialization, New CEO

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Martedì, Maggio 7, 2024
Biotechnology, General Health, Other Health, Health, Health Technology, FDA, Gene therapy, Inc., Partnership, Collection, CDMO, Therapy, Growth

As part of the change new CEO Thomas VanCott, Ph.D., will step in to lead the company, while David Wellis, Ph.D., will transfer to Head of Strategic Partnerships.

Key Points: 
  • As part of the change new CEO Thomas VanCott, Ph.D., will step in to lead the company, while David Wellis, Ph.D., will transfer to Head of Strategic Partnerships.
  • Excellos declined to disclose the origin of the multimillion-dollar investment, but according to CEO Dr. VanCott, it was provided by one of the current investors who expressed enthusiasm for Excellos' expansion of the business team.
  • It resolves one of the main scaling challenges at Excellos where the CEO role was hands-on for both operations and business development” stated Dr. VanCott.
  • “Excellos is now positioned to further assist our cell therapy and development partners with well-characterized metabolic and effector function profiles from highly-customized donors.”

Alcyone Therapeutics Announces Appointment of Dr. Norbert Riedel as Chairman of the Board and Dr. Kathrin Meyer as Chief Scientific Officer and Head of Research & Development

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Mercoledì, Maggio 8, 2024
Doctor of Philosophy, University, MicroRNA, Gene therapy, Hospital, Central nervous system disease, Rett syndrome, Innovation, Jazz Pharmaceuticals, Research, Ohio State University, History, CNS, Baxter International, Therapy, Batten disease, Patient, Acquisition, Gene expression, Nationwide Children's Hospital, Central nervous system, Health, SMARD1, Growth, Medical device

LOWELL, Mass., May 8, 2024 /PRNewswire/ -- Alcyone Therapeutics, a clinical-stage biotechnology company pioneering next-generation precision genetic therapies for complex neurological conditions, today announces appointments of Norbert Riedel, Ph.D., a skilled and experienced biopharmaceutical executive, as the Chairman of its Board of Directors, and Kathrin Meyer, Ph.D., a leader in development and clinical translation of genetic therapies for the central nervous system (CNS), as Alcyone's new Chief Scientific Officer and Head of R&D.

Key Points: 
  • "I am delighted and honored to join the board of Alcyone Therapeutics," said Dr. Riedel.
  • I am very excited about the programs we have in development to address the significant unmet patient needs in several neurological indications."
  • "Developing neurological genetic therapies demands both a highly precise biological mechanism of action and effective delivery to the central nervous system.
  • Dr. Kathrin Meyer is a pioneer in the research and clinical development of novel therapies for the CNS.

Ginkgo Bioworks Announces Presentations at the Upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

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Lunedì, Maggio 6, 2024
Gene therapy, Abstract, UTR, Machine learning, RNA therapeutics, RNA, Cell, Ginkgo, B cell, Automation, DNA, Therapy, Gene, Society, Acquisition, Messenger, Annual general meeting, NK, Vaccine

Ginkgo will present three posters in cell therapy and give one oral presentation in mRNA therapeutics.

Key Points: 
  • Ginkgo will present three posters in cell therapy and give one oral presentation in mRNA therapeutics.
  • These presentations demonstrate the power of Ginkgo's platform to drive innovation in the discovery and development of genetic medicines.
  • The addition of Patch Bio's assets opens up additional commercial opportunities in gene therapy, cell therapy, and RNA therapeutics and has the potential to accelerate progress on current programs.
  • Information on the poster presentations and oral presentation are listed below, and the full abstracts are available on the ASGCT meeting website .

Nvelop Therapeutics Announces Members of Its Scientific Advisory Board

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Giovedì, Maggio 2, 2024
Health, Genetics, Other Science, Research, Science, Biotechnology, Harvard University, Danaher Corporation, Novartis, TIL, PACE, Howard Hughes Medical Institute, MIT, Gene therapy, Broad Institute, Gene editing, Duke University, Beam Therapeutics, Microbiology, Faculty, Harvard Medical School, Mammoth Biosciences, Therapy, Thomas Dudley Cabot, Aarhus University, Molecular genetics, Hospital, Arena, Biomedical engineering, RNA, Editas Medicine, Natural science, Bluebird bio, Kite Pharma, Torque, BioMarin Pharmaceutical, Patient, Gene, HSC, Massachusetts General Hospital, Growth, Pharmaceutical industry

Nvelop Therapeutics , a biotechnology company engineering programmable, non-viral vehicles for the in vivo delivery of therapeutic cargo, today announced the members of its scientific advisory board, bringing together a world-class group of experts from across the fields of gene therapy, gene editing and drug delivery.

Key Points: 
  • Nvelop Therapeutics , a biotechnology company engineering programmable, non-viral vehicles for the in vivo delivery of therapeutic cargo, today announced the members of its scientific advisory board, bringing together a world-class group of experts from across the fields of gene therapy, gene editing and drug delivery.
  • Keith Joung, M.D., Ph.D. : Dr. Joung is co-founder of Nvelop Therapeutics, and a pioneer in the development of targeted gene and epigenetic editing technologies.
  • He has co-founded multiple additional biotechnology companies, including Beam Therapeutics, Chroma Medicine, Editas Medicine, Pairwise Plants, SeQure Dx and Verve Therapeutics.
  • He is also the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics and Chroma Medicine.

ATLATL Scientific and Miltenyi Biotec: A Five-Year Partnership Driving Biotech Innovation in Singapore and Beyond

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Martedì, Aprile 30, 2024
Health, Medical Devices, Genetics, Research, Science, Pharmaceutical, Biotechnology, Gene therapy, MOU, Miltenyi Biotec, Partnership, Cell, Technology Center, Multimedia, Pharmaceutical industry

ATLATL Scientific and Miltenyi Biotec are celebrating a landmark five-year partnership, marking a journey of collaboration and innovation in the biotech sector.

Key Points: 
  • ATLATL Scientific and Miltenyi Biotec are celebrating a landmark five-year partnership, marking a journey of collaboration and innovation in the biotech sector.
  • View the full release here: https://www.businesswire.com/news/home/20240430526379/en/
    ATLATL Scientific and Miltenyi Biotec are celebrating a landmark five-year partnership (Photo: Business Wire)
    Since the inception of their partnership five years ago, ATLATL Scientific and Miltenyi Biotec have worked closely together to leverage their respective expertise and resources.
  • The partnership between ATLATL Scientific and Miltenyi Biotec was further solidified at AsiaBio 2024, where they co-sponsored the event as booth partners.
  • The partnership between ATLATL Scientific and Miltenyi Biotec represents a testament to the power of collaboration in driving scientific innovation.

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

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Lunedì, Marzo 18, 2024
Gastroenteritis, Genomics, Metabolism, Neutropenia, Brain, Nerve, Mutation, RPD, Disease, Incidence, Partnership, Central nervous system, Genetics, GSK, Stomatitis, Patient, Research, Fats, PRV, Traffic barrier, Child, Viral, Gene therapy, SAN, Genome, Urinary tract infection, Rash, Hepatomegaly, Priority review, Stem cell, Infection, Therapy, MLD, Kyowa Kirin, Febrile neutropenia, Fever, PSLI, Prognosis, Health, Family, FDA, San Raffaele Hospital, ARSA, Orchard, Food, Pharmaceutical industry

“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.

Natalia Gomez-Ospina, MD, PhD is the Recipient of the 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award from the ACMG Foundation for Genetic and Genomic Medicine

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Mercoledì, Marzo 13, 2024
Research, Partner, Brain, B cell, FACMG, Gaucher's disease, Assistant, Pediatric Research, American Journal, Patient, Medical genetics, DSM-IV codes, Glycogen storage disease, System, Young Scientist Award, Gene therapy, Doctor of Philosophy, Stanford University, Protein, Genome, American College, Mucopolysaccharidosis, Genomics, Society, Hospital, Genetic, Cell, Young, Genetic distance, Hurler syndrome, Lucile Packard Children's Hospital

BETHESDA, Md., March 13, 2024 /PRNewswire/ -- Natalia Gomez-Ospina, MD, PhD is the recipient of the ACMG Foundation for Genetic and Genomic Medicine's 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award—the "Watson Award"—named for the American College of Medical Genetics and Genomics first and longstanding executive director, Michael S. Watson, MS, PhD, FACMG.

Key Points: 
  • BETHESDA, Md., March 13, 2024 /PRNewswire/ -- Natalia Gomez-Ospina, MD, PhD is the recipient of the ACMG Foundation for Genetic and Genomic Medicine's 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award—the "Watson Award"—named for the American College of Medical Genetics and Genomics first and longstanding executive director, Michael S. Watson, MS, PhD, FACMG.
  • "I am honored to receive this award in recognition of my dedication to advancing genetic and genomic medicine and my commitment to making a positive difference in the lives of patients with genetic diseases.
  • This award affirms the need for innovative approaches to treat such diseases and the immense potential that genome editing has in this regard.
  • "Dr. Gomez-Espina is a promising physician-scientist whose work is befitting the Dr. Michael Watson Genetic and Genomic Medicine Innovation Award, an award that honors an individual whose work has had a significant impact on genetic and genomic medicine.

MitoRx Therapeutics Strengthens Scientific Advisory Board with Appointment of Four International Experts

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Giovedì, Marzo 7, 2024
Therapy, CNRS, Target, Newcastle University, Facial muscles, Biotechnology, Professor, Teaching hospital, SAB, Neuromuscular disease, Ageing, Leiden University Medical Center, HD, DMD, Duchenne muscular dystrophy, Inflammation, LUMC, Mitochondrion, Gene therapy, Scientific method, Genetics, Knowledge, Leibniz Association, Publication, Translational research, Respiratory disease, Research, COPD, Pharmaceutical industry

OXFORD, United Kingdom, March 07, 2024 (GLOBE NEWSWIRE) -- MitoRx Therapeutics (MitoRx), a biotechnology company developing novel therapeutics that reverse mitochondrial dysfunction to arrest the progression of degenerative diseases, announces the appointment of four distinguished international experts to its Scientific Advisory Board (SAB): Caroline Le Guiner PhD, Milos Filipovic PhD, Professor Phil Hansbro PhD, and Professor Annemieke Aartsma-Rus.

Key Points: 
  • OXFORD, United Kingdom, March 07, 2024 (GLOBE NEWSWIRE) -- MitoRx Therapeutics (MitoRx), a biotechnology company developing novel therapeutics that reverse mitochondrial dysfunction to arrest the progression of degenerative diseases, announces the appointment of four distinguished international experts to its Scientific Advisory Board (SAB): Caroline Le Guiner PhD, Milos Filipovic PhD, Professor Phil Hansbro PhD, and Professor Annemieke Aartsma-Rus.
  • Dr. Jon Rees, Chief Executive Officer of MitoRx, said, “We are thrilled to welcome Dr.
  • Le Guiner, Dr. Filipovic, Professor Hansbro, and Professor Aartsma-Rus, to our Scientific Advisory Board.
  • She has a visiting professorship at the John Walton Muscular Dystrophy Research Center at the UK’s Newcastle University.

Fortress Biotech and Cyprium Therapeutics Announce $4.1 Million Grant from NINDS to Further Development of AAV-ATP7A Gene Therapy for Menkes Disease

Retrieved on: 
Lunedì, Marzo 4, 2024
Bangladesh Technical Education Board, Natural history, Gene, Nationwide, NIH, ATPase, FDA, Fortress Biotech, Food, Cerebrospinal fluid, Hospital, Principal investigator, Gene therapy, Human, Research institute, Therapy, Menkes disease, Patient, X-linked recessive inheritance, Cyprium, CSF, Clinical trial, Pediatrics, Fortification, Male, Genetics, Mutation, Spinal muscular atrophy, Copper, NDA, Orphan, AAV, DSM-IV codes, Research, Mouse, Pharmaceutical industry

MIAMI, March 04, 2024 (GLOBE NEWSWIRE) -- Cyprium Therapeutics, Inc. (“Cyprium”), a majority-owned subsidiary of Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), today announced that the National Institute of Neurological Disorders and Stroke (“NINDS”) of the National Institutes of Health (“NIH”) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus (“AAV”)-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease.

Key Points: 
  • Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase.
  • “By combining CUTX-101 with working copies of ATP7A delivered by AAV, we hope to enhance clinical outcomes in Menkes disease, a fatal rare pediatric disease.
  • Preclinical studies have demonstrated a synergistic effect of AAV-ATP7A and CUTX-101 in a reliable mouse model of Menkes disease.
  • In early studies, cerebrospinal fluid (“CSF”)-directed AAV gene therapy rescued 22-53% of mice with a mutation in the human Menkes disease homolog (mottled-brindled) when combined with CSF or subcutaneous copper.

Global Advanced Therapy Medicinal Products CDMO Market Size, Share & Trends Analysis Report 2024 - ResearchAndMarkets.com

Retrieved on: 
Martedì, Febbraio 27, 2024
Biotechnology, Health, Genetics, Stem Cells, Pharmaceutical, Therapy, Incidence, Tissue engineering, EMA, Regenerative medicine, USD, FDA, Partnership, ATMP, Cell, CDMO, Gene therapy, COVID-19, Disorder, Patient, Clinical trial, CureVac, CureVac COVID-19 vaccine, Tissue, Research, Pharmaceutical industry

The "Global Advanced Therapy Medicinal Products CDMO Market Size, Share & Trends Analysis Report by Product (Gene Therapy, Cell Therapy), Phase, Indication, Region, and Segment Forecasts, 2024-2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Advanced Therapy Medicinal Products CDMO Market Size, Share & Trends Analysis Report by Product (Gene Therapy, Cell Therapy), Phase, Indication, Region, and Segment Forecasts, 2024-2030" report has been added to ResearchAndMarkets.com's offering.
  • The global advanced therapy medicinal products CDMO market is expected to reach USD 18.8 billion by 2030 and is expected to register a CAGR of 18.92% from 2024 to 2030, owing to rising clinical trials for advanced therapy medicinal products and the increasing awareness among researchers about the benefits of advanced therapies, driving the advanced therapy medicinal products (ATMP) CDMO market growth.
  • Similarly, gene and cell therapy are attracting a lot of patients for the treatment of rare diseases, whose incidence is rising globally.
  • In January 2020, the agency released six final guidelines on the manufacturing and clinical development of safe & efficient gene therapy products.