ARSA

Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy

Retrieved on: 
Wednesday, March 20, 2024
Encephalitis, ICER, Bone marrow, Patient, DSM-IV codes, Urinary tract infection, Travel, Child, Health technology assessment, Therapy, Children's Hospital of Philadelphia, Central nervous system, Risk, MLD, Qualification, Health, WAC, NBS, Nerve, Mutation, UCSF, Disease, Federation, Research, Acquisition, Genome, Rash, Stem cell, HSC, Hospital, Family, Swelling, FDA, Diagnosis, HTA, Caregiver, Traffic barrier, Viral, Fever, Kyowa Kirin, PSLI, Liver, Government, San Raffaele Hospital, ARSA, Pharmaceutical industry

TOKYO, LONDON and BOSTON, March 20, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the details of its U.S. commercial launch of Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, the first FDA-approved therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile, (PSEJ), or early symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

Key Points: 
  • Prior to Lenmeldy, there were no treatment options in the U.S. for early-onset MLD beyond supportive and end-of-life care.
  • This approach has the potential to restore enzymatic function to stop or slow disease progression with a single treatment.
  • Utilizing results from such studies, a multi-stakeholder working group is finalizing a nomination to add MLD to the U.S.
  • Based on current timelines and assumptions, Orchard Therapeutics expects the nomination will be submitted in mid-year 2024.

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.

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Retrieved on: 
Monday, March 18, 2024
Encephalitis, Locomotion, Brain, B cell, Bone marrow, Cell, Dietary supplement, Nervous system, Risk, Patient, Public, Urinary tract infection, Animal, Metachromasia, DSM-IV codes, Peripheral nervous system, Viral, Human services, Hope, Death, Quality of life, Atidarsagene autotemcel, Rash, Therapy, Metachromatic leukodystrophy, Natural history, Fever, MLD, Heated tobacco product, Safety, Disability studies, Cosmetics, Health, Vaccine, Food, Swelling, FDA, Liver, Multimedia, ARSA, Pharmaceutical industry

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.

Key Points: 
  • Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.
  • The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene.
  • In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children.
  • Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy

Retrieved on: 
Thursday, January 25, 2024
Death, Pharmaceutical policy, Patient, MHRA, Therapy, Busulfan, Health care, Civil service commission, European Commission, Health, Mutation, Food, MLD, Central nervous system, Traffic barrier, ARSA, FDA, Priority review, Genome, HSC, Nerve, PDUFA, Orchard, Pharmaceutical industry

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “We are pleased to have reached an agreement with the Beneluxa consortium that enables sustainable access to treatment.
  • Multiple eligible MLD patients are currently in the treatment process and expected to receive Libmeldy in the coming months.

Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD

Retrieved on: 
Monday, December 11, 2023
Nerve, MLD, Traffic barrier, European Commission, Food, Agency, Priority review, Health care, Death, FDA, PDUFA, Civil service commission, HSC, MHRA, Therapy, Busulfan, ARSA, Mutation, Atidarsagene autotemcel, Genome, Central nervous system, Patient, Health, Pharmaceutical industry

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients’ own HSCs.
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “Today’s approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.

Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting

Retrieved on: 
Tuesday, May 16, 2023
Inhibitor, Neutralizing antibody, Hemolysis, Gene, NOD, MLD, PAH, Serum, SCID, Phenylketonuria, Trabecular meshwork, Gene editing, Gene expression, FIX, Publication, Metachromatic leukodystrophy, Messenger, PKU, Future, Administration, Immunosuppression, Genome, Intravitreal administration, Homologous recombination, Treatment, Vivisection, Immune system, PNH, Cell, Retinal pigment epithelium, NHP, Poster, RPE, Detection, Identification, Virus, ARSA, Society, Vaccine, Pharmaceutical industry, Truffle, Mouse, IND, C5

BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.

Key Points: 
  • These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.
  • Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates.
  • These data support the immunosuppression regimen used in Homology’s ongoing gene editing and gene therapy clinical trials.
  • In the poster, “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Optimization of HMI-202 Leading to HMI-204 Nomination,” Homology highlighted preclinical data on its optimized HMI-204 gene therapy candidate for MLD.

Advanced Reconstructive Surgery Alliance (ARSA) Announces New Partnership with CimiSurgical

Retrieved on: 
Monday, April 10, 2023
Learning, Partnership, Surgeon, Red Bank, Craniosynostosis, MBA, Moebius, Möbius syndrome, MD, Patient, Plastic, Education minister, Growth, Health, Plagiocephaly, Family, ARSA, Cleft lip and cleft palate, Dentistry

RED BANK, N.J., April 10, 2023 /PRNewswire-PRWeb/ -- Today, the Advanced Reconstructive Surgery Alliance ("ARSA") welcomes CimiSurgical to its National consortium. With the addition of CimiSurgical, ARSA adds a premier partner in New Jersey and further expands its footprint in the northeast region of the country.

Key Points: 
  • CimiSurgical Joins World-Class Network of Plastic and Reconstructive Surgeons, Adding to its Fast-Growing Roster
    RED BANK, N.J., April 10, 2023 /PRNewswire-PRWeb/ -- Today, the Advanced Reconstructive Surgery Alliance ("ARSA") welcomes CimiSurgical to its National consortium.
  • With the addition of CimiSurgical, ARSA adds a premier partner in New Jersey and further expands its footprint in the northeast region of the country.
  • They are also well versed and esteemed in the areas of breast surgery, hand surgery, skin pathology, and Mohs reconstruction.
  • "CimiSurgical is one of the premier pediatric and craniofacial reconstruction practices in the country," said ARSA CEO, Andrew Elkwood, MD, MBA.

Homology Medicines Presents Preclinical Data Supporting Immunosuppression Regimen in Ongoing PKU and Hunter Syndrome Clinical Trials, and Details Optimized MLD Gene Therapy Candidate at the 19th Annual WORLDSymposium™ Meeting

Retrieved on: 
Wednesday, February 22, 2023
Clinical trial, Dexamethasone, Central nervous system, ARSA, Administration, WORLD, MPS II, Brain, Research, PAH, Publication, Phenylketonuria, CNS, Metachromasia, PKU, Gene expression, MPS, JumpStart, Messenger, NAB, MLD, Hunter syndrome, Vaccine

BEDFORD, Mass., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data that supports the targeted, prophylactic immunosuppression regimen in the ongoing pheEDIT gene editing clinical trial in adults with phenylketonuria (PKU) and juMPStart gene therapy trial in adults with Hunter syndrome (MPS II). Homology also shared additional details of the optimized, in vivo gene therapy candidate HMI-204 for metachromatic leukodystrophy (MLD) during the 19th Annual WORLDSymposium™ Meeting.

Key Points: 
  • Homology also shared additional details of the optimized, in vivo gene therapy candidate HMI-204 for metachromatic leukodystrophy (MLD) during the 19th Annual WORLDSymposium™ Meeting.
  • administration in the murine disease model and resulted in levels of enzyme activity predicted to lead to efficacy in vivo.
  • In the poster titled, “Gene Therapy for Metachromatic Leukodystrophy: Lead Candidate Optimization,” a single I.V.
  • Additionally, packaging productivity of HMI-204 demonstrated a substantial improvement in vector genome yields, compared to the earlier MLD gene therapy candidate.

Advanced Reconstructive Surgery Alliance (ARSA) Announces New Partnership with Plastic, Reconstructive, and Microsurgical Associates (PRMA)

Retrieved on: 
Wednesday, January 11, 2023
LTP, Learning, PAP, Surgeon, GAP, SIEA, Partnership, ARSA, Growth, Plastic surgery, Plastic, Ministry of Education (Malaysia), Breast reconstruction, Breast cancer, Health, Dentistry, MD

TRINTON FALLS, N.J., Jan. 11, 2023 /PRNewswire-PRWeb/ -- Today, the Advanced Reconstructive Surgery Alliance ("ARSA") welcomes "PRMA" to its national consortium. With the addition of PRMA, ARSA adds a key partner in the southern region of the country and continues to work toward its goal of becoming a nation-wide provider.

Key Points: 
  • TRINTON FALLS, N.J., Jan. 11, 2023 /PRNewswire-PRWeb/ -- Today, the Advanced Reconstructive Surgery Alliance ("ARSA") welcomes " PRMA " to its national consortium.
  • "The surgeons here at PRMA are thrilled to join our colleagues at Advanced Reconstructive Surgery Alliance (ARSA)," stated Minas Chrysopoulo, MD, President of PRMA.
  • The surgeons at PRMA have performed thousands of breast reconstructions, including over 11,000 microsurgical flap procedures, making PRMA one of the leading centers in the world.
  • If interested in learning more about a partnership with ARSA, contact:

Orchard Therapeutics Announces Swissmedic Validation of the Marketing Authorization Application for Libmeldy (atidarsagene autotemcel)

Retrieved on: 
Thursday, December 1, 2022
United Kingdom, Literature, Australia New Zealand Therapeutic Products Authority, Therapy, Brain, Social media, Agency, Medication package insert, Risk, Patient, GSK, HSC, Late, Union, Infant, Dementia, ARSA, Royal commission, DOI, SAN, Systematic review, Nervous system, Seizure, Spasticity, MAA, Bone marrow, EMA, Partnership, Mortality, European Commission, Disease, Gallbladder, Liver, Safety, Multiple birth, Spleen, Mutation, Gene, MLD, File, Pharmaceutical industry, Child, Metachromatic leukodystrophy, EU, Orchard

The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020.

Key Points: 
  • The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020.
  • Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures.
  • For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
  • At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases.