Mannose 6-phosphate

M6P Therapeutics Presented Data on M041, A Recombinant Enzyme Therapy, for the Treatment of Sanfilippo B Syndrome at MPS 2021

Retrieved on: 
Wednesday, July 28, 2021

These data illustrate M041s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.

Key Points: 
  • These data illustrate M041s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.
  • There are currently no approved therapies for Sanfilippo B syndrome, also known as MPS IIIB.
  • Sanfilippo B syndrome is characterized by a defect in the NAGLU gene providing instructions for producing the enzyme alpha-N-acetylglucosaminidase.
  • M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).

M6P Therapeutics Announces Presentation at the Upcoming 16th International Symposium of MPS and Related Diseases

Retrieved on: 
Wednesday, July 21, 2021

M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced today its participation at the 16th International Symposium of MPS and Related Diseases, a research conference dedicated to the exchange of knowledge on mucopolysaccharidoses (MPS) and related syndromes, taking place virtually July 23-25, 2021.

Key Points: 
  • M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced today its participation at the 16th International Symposium of MPS and Related Diseases, a research conference dedicated to the exchange of knowledge on mucopolysaccharidoses (MPS) and related syndromes, taking place virtually July 23-25, 2021.
  • Details related to M6PTs oral presentation at MPS 2021 are below:
    M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).
  • This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases.
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics to Host Key Opinion Leader Webinar on Lysosomal Storage Disorders

Retrieved on: 
Monday, July 19, 2021

M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced that it will host a key opinion leader (KOL) webinar on LSDs on Wednesday, July 28, 2021 at 10:00 a.m.

Key Points: 
  • M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced that it will host a key opinion leader (KOL) webinar on LSDs on Wednesday, July 28, 2021 at 10:00 a.m.
  • LSDs are a family of approximately 50 rare, genetic, and life-threatening diseases characterized by a deficiency in a specific lysosomal enzyme.
  • M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics to Participate in Upcoming Fireside Chat Discussing the Company’s First-in-Class Platform for Lysosomal Enzyme and Gene Delivery at the ROTH Virtual Private Company Forum

Retrieved on: 
Friday, June 25, 2021

The event will consist of Virtual Panels on Emerging Therapeutics and select Fireside Chats with ROTH Research Analysts.

Key Points: 
  • The event will consist of Virtual Panels on Emerging Therapeutics and select Fireside Chats with ROTH Research Analysts.
  • For more information about the fireside chat, register here or contact your ROTH representative.
  • M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics Presents M002 Gene Therapy Preclinical Proof-of-Concept Data for the Treatment of Mucolipidosis Type II at the ASGCT 24th Annual Meeting

Retrieved on: 
Tuesday, May 11, 2021

Krysiak continued, \xe2\x80\x9cTogether, these results provide a strong foundation for our broader gene therapy pipeline, which leverages our S1S3 bicistronic platform to enhance M6P content across lysosomal enzymes.

Key Points: 
  • Krysiak continued, \xe2\x80\x9cTogether, these results provide a strong foundation for our broader gene therapy pipeline, which leverages our S1S3 bicistronic platform to enhance M6P content across lysosomal enzymes.
  • The mutation leads to GlcNAc-1-phosphotransferase deficiency and inability to phosphorylate (add mannose 6-phosphate, or M6P) lysosomal enzymes.
  • This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases.
  • M6P Therapeutics\xe2\x80\x99 mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics Announces Presentation at the Upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Retrieved on: 
Wednesday, April 28, 2021

M6P Therapeutics\xe2\x80\x99 proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both recombinant enzyme and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product.

Key Points: 
  • M6P Therapeutics\xe2\x80\x99 proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both recombinant enzyme and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product.
  • This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases.
  • M6P Therapeutics\xe2\x80\x99 team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform.
  • M6P Therapeutics\xe2\x80\x99 mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics Presents Promising Preclinical Data in Lysosomal Storage Disorders at the 17th Annual WORLDSymposium™ 2021

Retrieved on: 
Thursday, February 11, 2021

M6P Therapeutics , a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced today the presentation of promising preclinical data at the 17th Annual WORLDSymposium, a research conference dedicated to lysosomal diseases being held virtually Feb. 8-12, 2021.

Key Points: 
  • M6P Therapeutics , a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced today the presentation of promising preclinical data at the 17th Annual WORLDSymposium, a research conference dedicated to lysosomal diseases being held virtually Feb. 8-12, 2021.
  • In two separate poster presentations, M6P Therapeutics researchers report preclinical efficacy and safety results for Gaucher disease and mucolipidosis II (MLII) respectively.
  • M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board

Retrieved on: 
Wednesday, February 3, 2021

The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.

Key Points: 
  • The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.
  • Her research has defined the molecular basis for the recognition of lysosomal enzymes by the M6P receptors.
  • M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics Receives Six Rare Pediatric Disease Designations from the U.S. FDA for Company’s Deep Pipeline of Programs for Lysosomal Storage Disorders

Retrieved on: 
Thursday, January 28, 2021

In addition, the FDA granted two orphan drug designations (ODDs) for its gene therapy programs for Gaucher disease and mucolipidosis.

Key Points: 
  • In addition, the FDA granted two orphan drug designations (ODDs) for its gene therapy programs for Gaucher disease and mucolipidosis.
  • M6P Therapeutics is developing an innovative technology that regulates the natural mechanism for trafficking enzymes to lysosomes.
  • The companys bicistronic-S1S3 platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.
  • We are grateful to the FDA for these multiple orphan and rare pediatric disease designations that highlight the need for new and improved therapies to address unmet needs.