Enzyme replacement therapy

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Death, Glycosaminoglycan, B cell, Enzyme, Mutation, Medicine, Cell, Mental disability, Disease, Patient, Committee, Knowledge, Scheie syndrome, Regulation, EMA, IRIS, Periodic breathing, Mucopolysaccharidosis, European, COMP, Enzyme replacement therapy, European Commission, Safety, DSM-IV codes, IQVIA, Marketing, Haematopoiesis, Liver, RDS, FGK, Hurler syndrome, MPS, Pharmaceutical industry

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Key Points: 


Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Fabry disease, Brain, Cell, CD34, Disease, Nervous system, Patient, Committee, Knowledge, Tissue, Stroke, Kidney failure, Fats, Regulation, Autotransplantation, EMA, IRIS, Human, Eye, Sugar, European, Skin, COMP, Heart, Sponsor, European Commission, Enzyme replacement therapy, Blood, PPD, Pain, Safety, Marketing, Bone marrow, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Jean-Jacques Bienaimé Appointed Chairman of the Board at Owkin

Retrieved on: 
Monday, March 25, 2024
Research, Technology, Health Technology, Software, Biotechnology, Pharmaceutical, Health, Science, Artificial Intelligence, Other Science, Growth, Information technology, BioMarin Pharmaceutical, Intelligence, École, University, M.B.A, Biotechnology Innovation Organization, MD, Medicine, Commerce, Enzyme replacement therapy, Multimedia, Economics

Owkin , a TechBio that combines the best of human and artificial intelligence to find the right treatment for every patient, is very pleased to announce the appointment of Jean-Jacques Bienaimé as Chairman to its Board of Directors.

Key Points: 
  • Owkin , a TechBio that combines the best of human and artificial intelligence to find the right treatment for every patient, is very pleased to announce the appointment of Jean-Jacques Bienaimé as Chairman to its Board of Directors.
  • “We are thrilled to have Jean Jacques Bienaimé join our Board of Directors as the new Chairman,” said Thomas Clozel, MD, co-founder and CEO of Owkin.
  • Mr. Bienaimé studied economics at the Ecole de Commerce de Paris and received an M.B.A from the University of Pennsylvania.
  • Mr. Bienaimé is joining a board of directors led by experienced figures from the biopharmaceutical industry and leading venture capital funds.

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

The United Mitochondrial Disease Foundation Announces Inaugural Venture Philanthropy Investment; The Mito Fund Invests $500k in Pierrepont Therapeutics, Inc.

Retrieved on: 
Thursday, April 4, 2024
Disease, Encephalopathy, Risk, Trustee, Patient, MNGIE, Research, Columbia University, Mitochondrial disease, Science, Death, MD, Malnutrition, Enzyme replacement therapy, UMDF, Natural history, Pharmaceutical industry

PITTSBURGH, April 4, 2024 /PRNewswire-PRWeb/ -- The United Mitochondrial Disease Foundation (UMDF) today announced the inaugural investment for its venture philanthropy initiative, The Mito Fund.

Key Points: 
  • PITTSBURGH, April 4, 2024 /PRNewswire-PRWeb/ -- The United Mitochondrial Disease Foundation (UMDF) today announced the inaugural investment for its venture philanthropy initiative, The Mito Fund.
  • "We've said all along that time is something mitochondrial disease families just don't have, especially when it comes to something as devastating as MNGIE.
  • Following an extensive review process, the Pierrepont investment was recommended by UMDF's Venture Philanthropy Investment Committee, an advisory group consisting of world-class experts in the areas of mitochondrial disease, pharmaceutical development, and venture investments.
  • UMDF thanks the Kallaos and Wright families for their founding gifts that helped make the launch of The Mito Fund possible.

PANTHERx®Rare Selected by Edenbridge Pharmaceuticals as Specialty Pharmacy for Yargesa® (miglustat 100 mg capsules)

Retrieved on: 
Monday, April 1, 2024
Disease, Capsule, Patient, Pharmacy, Bone pain, Hepatomegaly, Enzyme replacement therapy, Anemia, Gaucher, Thrombocytopenia, Liver, Spleen, Pharmaceutical industry

Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.

Key Points: 
  • Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.
  • Through this process, Yargesa® reduces the buildup of harmful glycosphingolipids and symptoms of the disease.
  • This accumulation leads to manifestations including but not limited to enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.
  • "We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease.

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

Retrieved on: 
Wednesday, February 14, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Enzyme replacement therapy, BBB, JCR, MPS II, Traffic barrier, Glycogen storage disease, Ministry, MHLW, ERT, Hunter syndrome, Syndrome, Scheie syndrome, MPS, Safety, Developmental psychology, Hurler syndrome, Child, Patient, Hurler–Scheie syndrome, Trial of the century, Consciousness, DSM-IV codes, Hypotension, Mouse, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

Retrieved on: 
Wednesday, February 14, 2024
Enzyme replacement therapy, Fabry disease, ICV, EMA, Intelligence, European, Glycolipid, ERT, Sanfilippo syndrome, Hunter syndrome, Death, MPS, Prevalence, ODD, Safety, Sanfilippo, IDS, Disorder, Patient, Hanmi Pharmaceutical, Fibrosis, Clinical trial, Knowledge, Kidney disease, Disease, Urine, Heart, Alpha-galactosidase, Phase, Male, Kidney, Glycogen storage disease, Brain, Orphan, Mouse, Pharmaceutical industry

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

Key Points: 
  • YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.
  • WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.
  • In 2012, GC Biopharma succeeded in developing the world's second treatment for Hunter syndrome, "Hunterase" solely using domestic technology.
  • GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

Sangamo Therapeutics Announces U.S. FDA Alignment on Abbreviated Pathway to Potential Approval and EMA Prime Eligibility for ST-920 in Fabry Disease

Retrieved on: 
Monday, February 12, 2024
Research, FDA, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Fabry disease, EMA, Orphan drug, Type D, Investment, Female, ERT, BLA, STAAR, Therapy, PRIME, Sangamo Therapeutics, Patient, Male, Enzyme replacement therapy, Partnership, Pharmaceutical industry

The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm.

Key Points: 
  • The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm.
  • A head-to-head comparison with Enzyme Replacement Therapy (ERT) is not part of the proposed study design deemed acceptable by the FDA.
  • This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated.
  • Sangamo is deferring additional investments in planning for a registrational trial until a collaboration partnership is secured.

Chiesi Global Rare Diseases Presents Long-Term Data on Treatment with Velmanase Alfa in Alpha-Mannosidosis and Announces Additional Presentations at the 20th Annual WORLDSymposium™ Research Meeting

Retrieved on: 
Thursday, February 8, 2024
Conjunctivitis, Arthralgia, Anaphylaxis, Common, ADAS, Birth, Prevalence, Velmanase alfa, Fabry disease, Human, Diagnosis, Headache, Cough, Erythema, ERT, Glycogen storage disease, Alpha-mannosidosis, DSM-IV codes, Vomiting, Hospital, Incidence, Patient, Rash, Nursing, Data, Chills, Enzyme replacement therapy, Pharmaceutical industry

BOSTON, Feb. 8, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the presentation of long-term data from up to 12 years of treatment with velmanase alfa in patients with alpha-mannosidosis (AM). The company also announced the presentation of immunogenicity and tolerability data from its pegunigalsidase alfa clinical trial program in Fabry disease, as well as analyses of the prevalence, diagnosis, and progression of these rare lysosomal storage disorders. The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California.

Key Points: 
  • The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California.
  • Several poster presentations are highlighting immunogenicity and tolerability data from the pegunigalsidase alfa clinical trial program in Fabry disease.
  • The analysis includes 108 patients, 27 (25%) of whom were ADA+ before receiving their first dose of pegunigalsidase alfa.
  • There was a low incidence of de novo ADAs (n=17) occurring predominantly in the first year of treatment with pegunigalsidase alfa.