M6P

M6P Therapeutics Presents Promising Preclinical Data In Lysosomal Storage Disorders At The 19th Annual WORLDSymposium™ 2023

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Tuesday, February 21, 2023
Gaucher's disease, Glycogen storage disease, Mannose, Glycogen storage disease type II, Abstract, Q&A, Tissue, M6P, Cell, Research, Therapy, LSD, Gaucher, Pharmaceutical industry

ST. LOUIS, Mo., Feb. 21, 2023 (GLOBE NEWSWIRE) -- M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of promising preclinical data at the 19th Annual WORLDSymposium™, a research conference dedicated to lysosomal diseases being held in Orlando, FL, and virtually Feb. 22-26, 2023.

Key Points: 
  • ST. LOUIS, Mo., Feb. 21, 2023 (GLOBE NEWSWIRE) -- M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of promising preclinical data at the 19th Annual WORLDSymposium™, a research conference dedicated to lysosomal diseases being held in Orlando, FL, and virtually Feb. 22-26, 2023.
  • In five poster presentations, including two Contemporary Forum presentations, M6P Therapeutics’ researchers report preclinical efficacy results for LSDs, including Gaucher disease and Pompe disease.
  • “We are thrilled with the continued development of our S1S3 phosphotransferase platform for producing lysosomal enzymes with high levels of mannose 6-phosphate to enable much better drug targeting and delivery of therapeutic lysosomal enzymes to all affected cells and tissues,” said Cuong Do, Chairman of M6P Therapeutics.
  • The abstracts will also be made available on the “ Publications ” section of M6PT’s corporate website following the presentations.

Amicus Therapeutics Receives Positive CHMP Opinion for Pombiliti™ (cipaglucosidase alfa) for Late-Onset Pompe Disease

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Friday, December 16, 2022
Lysosome, European Medicines Agency, European Commission, CHMP, Physician, Cell, NAS, EMA, Muscle, Annual report, Glycogen storage disease type II, BIS, Trial of the century, M6P, ERT, Amicus Therapeutics, Tissue, Glycogen storage disease, PROPEL, Infant, Glycogen, Committee for Medicinal Products for Human Use, Committee, Patient, EC, Disease, Therapy, Royal commission, Enzyme replacement therapy, Degenerative disease, Blood, Miglustat, Compte rendu, GAA, Skeletal muscle, Vaccine, Pharmaceutical industry, Dietary supplement, EU, Neurology

PHILADELPHIA, Dec. 16, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization of cipaglucosidase alfa, a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). A decision from the European Commission (EC) on cipaglucosidase alfa, the enzyme replacement therapy component of AT-GAA, is expected in the first quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI™. The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023.

Key Points: 
  • The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023.
  • Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA).
  • Disease severity ranges on a spectrum, but predominant manifestations are skeletal muscle weakness and progressive respiratory involvement.
  • “Today’s positive CHMP opinion for Pombiliti™ (cipaglucosidase alfa) is a significant milestone and major step towards bringing this much needed new treatment for all adults living in the EU with late-onset Pompe disease.

M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress

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Tuesday, October 11, 2022
Health, Other Science, General Health, Research, Science, Biotechnology, Congress, ESGCT, LSD, Therapy, Conference, Animal, Cell, Drug development, Gene, Publication, Glycogen storage disease, Tissue, Program, European Society of Gene and Cell Therapy, Technology, R, Gaucher's disease, Edinburgh International Conference Centre, Neuron, AAV, Phosphorylation, Metabolic disorder, Glycosphingolipid, CEO, A-DNA, CNS, Brain, Â, Mannose, GBA1, Central nervous system, M6P, Poster, Pharmaceutical industry, Fine chemical, Gaucher

The Congress takes place in-person and virtually in Edinburgh, Scotland from Oct. 11-14, 2022 at the Edinburgh International Conference Centre.

Key Points: 
  • The Congress takes place in-person and virtually in Edinburgh, Scotland from Oct. 11-14, 2022 at the Edinburgh International Conference Centre.
  • The data from this preclinical study illustrate the potential of our AAV-GBA-S1S3 gene therapy candidate for the treatment of Gaucher disease, including neuronopathic phenotypes, said Pawel Krysiak, President and CEO of M6P Therapeutics.
  • M012 is part of our pipeline of candidates leveraging a first-in-class co-expression S1S3 platform technology for the treatment of lysosomal storage disorders.
  • Gene therapy could be a potential therapeutic approach to treat all three types of Gaucher disease.

Worldwide Specialty Injectable Industry to 2028 - Size, Share, Outlook, and Opportunity Analysis - ResearchAndMarkets.com

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Wednesday, May 18, 2022
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The "Specialty Injectable Market, by Drug Type, by Application, by Distribution Channel, and by Region - Size, Share, Outlook, and Opportunity Analysis, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Specialty Injectable Market, by Drug Type, by Application, by Distribution Channel, and by Region - Size, Share, Outlook, and Opportunity Analysis, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.
  • Specialty injectable is a high cost drug therapy that requires administration by injection.
  • Specialty injectable are high cost drugs that require precise temperature control, special handling, and clinical management to ensure the drug performance.
  • This is expected to drive the global specialty injectable market growth over the forecast period.

M6P Therapeutics to Present at the 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis

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Friday, October 8, 2021
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Linda, Person, Epilepsy, Endocytosis, N, Nervous system, Therapy, LSD, Dementia, CLN3, Plasmid, Endoglycosidase H, Batten disease, A-DNA, Neuronal ceroid lipofuscinosis, CLN2, PNGase F, Glycogen storage disease, Drug development, Lysosome, Mannose, Neuron, Neurodegeneration, Washington University in St. Louis, Eric, M6P, Prevalence, Severe cognitive impairment, NCLS, Enzyme, Communication, International, NCL, Autosome, International Congress on Tuberculosis, Disease, Patient, Enzyme replacement therapy, TPP1, Research, Technology, Phosphorylation, Pharmaceutical industry, Fine chemical

Neuronal ceroid lipofuscinosis type 2 (CLN2) is a neurodegenerative disorder caused by autosomal recessive mutations in the TPP1 gene, leading to the deficiency of TPP1, a lysosomal enzyme.

Key Points: 
  • Neuronal ceroid lipofuscinosis type 2 (CLN2) is a neurodegenerative disorder caused by autosomal recessive mutations in the TPP1 gene, leading to the deficiency of TPP1, a lysosomal enzyme.
  • Batten disease originally referred specifically to the juvenile and most common form of neuronal ceroid lipofuscinosis (NCL), now known as CLN3 .
  • Most forms are inherited in an autosomal recessive manner; however, autosomal dominant inheritance has been reported in one adult-onset form (neuronal ceroid lipofuscinosis 4B).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.