Gene therapy

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

Retrieved on: 
Monday, March 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

Retrieved on: 
Wednesday, March 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

Retrieved on: 
Thursday, March 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: 
Monday, March 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: 
Tuesday, February 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.

City of Hope Research Featuring the Successful Treatment of the Oldest Patient to Achieve Remission for Leukemia and HIV Published in The New England Journal of Medicine (NEJM)

Retrieved on: 
Wednesday, February 14, 2024
Oncology, AIDS, Health, Infectious Diseases, Hospitals, Research, Science, Infection, Bone marrow, Cancer, Division, Immune system, Multimedia, Syndrome, Graft-versus-host disease, Acute leukemia, Blood, CCR5, Haematopoietic system, Transplant, Government, BMT, National Cancer Institute, Laboratory, B cell, Gene therapy, Medicare, Patient, Mutation, HIV, Dentistry

View the full release here: https://www.businesswire.com/news/home/20240214747351/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240214747351/en/
    "City of Hope Patient" Paul Edmonds with City of Hope doctors, Jana K. Dickter, M.D., and Monzr Al Malki, M.D.
  • Edmonds is also the person who had HIV the longest — for over 31 years — among these five patients.
  • Known as the “City of Hope patient” among these five patients, Edmonds received a transplant at City of Hope on Feb. 6, 2019, and is now considered to be cured of leukemia.
  • City of Hope has exceptional transplant outcomes year after year, according to the Center for International Blood & Marrow Transplant Research.

Dyne Therapeutics Announces Upcoming Presentations on Initial Clinical Data From its ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients at the 2024 Muscular Dystrophy Association Clinical & Scientific Conference

Retrieved on: 
Wednesday, February 14, 2024
Therapy, Nationwide, Principal, DYN, DMD, Duchenne muscular dystrophy, Conference, DM1, Gene therapy, Safety, Muscular Dystrophy Association, Patient, MPH, Asian literature, PMO, Myopathy, MDA, Episcopal conference, Pharmaceutical industry

WALTHAM, Mass., Feb. 14, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced two oral presentations at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida and virtually.

Key Points: 
  • Both presentations will highlight the initial clinical data from the ACHIEVE and DELIVER trials reported by Dyne in January 2024.
  • The initial clinical data from the ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) includes 6-month data from the 1.8 mg/kg (approximate ASO dose) cohort (n=16) and 3-month data from the 3.4 mg/kg Q4W cohort (n=16).
  • Initial 6-month clinical data from the 5 mg/kg (approximate PMO dose) cohort (n=6) of the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping will also be presented.
  • Additionally, both presentations will include safety data from multiple cohorts in ACHIEVE and DELIVER trials which was shared in January.

Accumulus Synergy Launches Highly Anticipated Cloud-Based Platform

Retrieved on: 
Thursday, February 8, 2024
World Health Organization, Agency, Q&A, Cell, Breast cancer, Organization, Roche, WHO, Gene therapy, Senior, Patient, AstraZeneca, Pharmaceutical industry

BURLINGAME, Calif., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Accumulus Synergy, Inc. (“Accumulus”) is proud to announce that it has launched the initial version of its first-in-kind information and data exchange platform.

Key Points: 
  • BURLINGAME, Calif., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Accumulus Synergy, Inc. (“Accumulus”) is proud to announce that it has launched the initial version of its first-in-kind information and data exchange platform.
  • “Today marks the beginning of a new era for the life sciences - regulatory ecosystem,” commented Frank Nogueira, Accumulus Chief Executive Officer.
  • “This release is the first step toward achieving our mission to dramatically accelerate critical therapies to citizens of the world.
  • “The launch of the platform moves the Accumulus mission from concept to reality.”
    To learn more about platform access and the different ways to engage with Accumulus Synergy, please email [email protected] .

Genenta Demonstrated Reprogramming of the Tumor Microenvironment in GBM Patients, Paving the Way for Innovative Treatments of Solid Tumors

Retrieved on: 
Thursday, February 8, 2024
GBM, Toxicity, CMO, RNA, Human, Gene therapy, Patient, San Raffaele, TME, Tumor microenvironment, Neoplasm, Stem cell, Pharmaceutical industry

The second patient has been enrolled and the treatment is planned.

Key Points: 
  • The second patient has been enrolled and the treatment is planned.
  • Thus far, the preliminary data indicate no dose-limiting toxicities related to Temferon have been detected in any of 22 treated patients.
  • Temferon-derived differentiated cells were evident within the peripheral blood 14 days after infusion and were still detectable at more than 24 months.
  • We expect reporting top line Phase 1 dose-ranging data by the end of 2Q24.

Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome

Retrieved on: 
Wednesday, January 31, 2024
FDA, Other Health, Pharmaceutical, Managed Care, General Health, Biometrics, Mental Health, Infectious Diseases, Neurology, Genetics, Clinical Trials, Biotechnology, Science, Other Science, Research, Health, Parent, Synapse, Neuroscience, NHP, Mutation, Spectrum, Genetic disorder, Quality of life, Learning, PMSF, Memory, ASD, PMS, ICV, Investigational New Drug, Cognitive science, Broad, Prevalence, IND, Jaguar, Genetic testing, Frasier, SHANK3, ID, Spinal cord, Haploinsufficiency, DSM-IV codes, Gene therapy, Patient, Central nervous system, Harvard University, Speech, Food, Neuron, Disease, Communication, Risk, Sibling, Disorder, Psychiatry, 22q13 deletion syndrome, Intellectual disability, Broad Institute, MIT, Pediatrics, Pharmaceutical industry

Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).

Key Points: 
  • Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).
  • JAG201 aims to deliver functional SHANK3 via the AAV9 vector to treat the root cause of the disease.
  • “We are pleased to receive FDA clearance to bring our investigational SHANK3 gene therapy to the clinic.
  • The pre-clinical data indicate that JAG201 may have the potential to be transformative for those suffering with the disorder,” said Joe Nolan, chief executive officer of Jaguar Gene Therapy.