News

 

Below please find the latest news at M6P Therapeutics.

Update from Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) on Mucolipidosis Type II

October 9, 2021 – The Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) announced that they will be able to initiate the Natural History studies on a model of mucolipidosis type II (ML II) in 2022. M6PT met with both the MCRN and Cure ML to provide an update on the status of M6PT’s preclinical development programs for MLII/III , and to discuss collaboration efforts to develop and foster the discovery and development of new therapies for ML.

 

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

ST. LOUIS, Mo., – October 8, 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 the Company’s participation at the 17th International Congress on Neuronal Ceroid Lipofuscinosis (NCLs), or NCL2021, being held from October 6-10, 2021, at the Eric P. Newman Education Center (EPNEC), on the Medical School Campus of Washington University in St Louis. Linda Zhao, a member of Dr. Lin Liu’s R&D team, will present the abstract titled, “TPP1 with increased mannose 6-phophate content and cell uptake for Neuronal ceroid lipofuscinosis type 2 disease” at NCL2021.

 

Dr. Stuart Kornfeld, M6P Therapeutics’ Co-Founder, to Receive the Roscoe O. Brady Award For Innovation and Accomplishment at WORLDSymposium™ 2022

ST. LOUIS, Mo., – September 30, 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 WORLDSymposium™ has named its co-founder, Stuart Kornfeld, MD, as the recipient of the 2022 Roscoe O. Brady Innovation and Accomplishment award. Each year, WORLDSymposium™ recognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. This award has been renamed the Roscoe O. Brady Award for Innovation and Accomplishment to honor one of the early pioneers in lysosomal research, Roscoe O. Brady, MD, a beloved mentor to many currently working in the field.

 

M6P Therapeutics KOL Webinar: July 28, 2021

The webinar features a fireside chat with KOLs Gregory Enns, M.D., Lucile Salter Packard Children’s Hospital Stanford School of Medicine, and Mark S. Sands, Ph.D., Departments of Medicine and Genetics at Washington University School of Medicine, who provided an overview of the current treatment landscape and unmet medical needs in LSDs, including Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidoses, and mucolipidoses. The webinar also features an update from the M6PT management team on its recombinant enzyme and gene therapy S1S3 bicistronic technology platform for the treatment of LSDs.

 

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

ST. LOUIS, Mo., – July 28, 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 pre-clinical proof-of-concept data on M041 in a poster presentation by Patricia Dickson, M.D., Centennial Professor of Pediatrics and Chief, Division of Genetics and Genomic Medicine, Washington University of St. Louis…

 

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

ST. LOUIS, Mo., – 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…

 

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

ST. LOUIS, Mo., – 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. ET…

 

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

ST. LOUIS, Mo., – June 25, 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 Pawel Krysiak, M6PT’s president and chief executive officer, will participate in a fireside chat at the ROTH Virtual Private Company Forum…

 

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

ST. LOUIS, Mo., – May 11, 2021 M6P Therapeutics, (“the Company”), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of preclinical proof-of-concept data for M002, its gene therapy candidate for mucolipidosis type II (MLII), at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting…

 

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

ST. LOUIS, Mo., – April 28, 2021 M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced today a poster presentation on mucolipidosis type II at the American Society of Gene & Cell Therapy’s (ASGCT) 24th Annual Meeting: May 11-14, 2021…

 

M6P Therapeutics Supports Rare Disease Day 2021 and Joins Global Movement to Increase Awareness of Rare Diseases

ST. LOUIS, Mo., – Feb. 11, 2021 M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its support for Rare Disease Day 2021 and commitment to the rare disease community. Rare Disease Day is an annual awareness day dedicated to elevating the public understanding of rare diseases and their impact on patients’ lives…

 

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

ST. LOUIS, Mo., – Feb. 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. In two separate poster presentations, M6P Therapeutics’ researchers report preclinical efficacy and safety results for Gaucher disease and mucolipidosis II (MLII) respectively…

 

M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board

ST. LOUIS, Mo., Feb. 3, 2021 M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its scientific advisory board (SAB) that will support the Company’s mission of translating its innovative bicistronic-S1S3 technology platform into best-in-class therapies that address unmet needs within the LSD community. The Company’s platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies…

 

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

ST. LOUIS, Mo., – Jan. 28, 2021 – M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced that the U.S. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs. In addition, the FDA granted two orphan drug designations (ODDs) for its gene therapy programs for Gaucher disease and mucolipidosis…