Discussions will focus on Pompe Disease and M6P’s S1S3 Co-expression Platform Technology

ST. LOUIS, Mo., April 09, 2025 (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 it will host a virtual key opinion leader (KOL) event on Tuesday, April 22, 2025 at 11:00 AM ET featuring Professor Gregory Enns (Stanford Medicine). To register, click here.

Professor Enns will provide expert insights into unmet need and current treatment landscapes for lysosomal storage disorders (LSDs) with a focus on Pompe disease, a rare, autosomal recessive, inherited disorder. He will be joined by Tiffany House, President of AMDA-Pompe and Vice-Chair of the International Pompe Association.

In addition, the event will highlight M6P's S1S3 co-expression platform technology, designed to enhance mannose 6-phosphate (M6P) content on lysosomal enzymes to increase lysosomal targeting to develop both enzyme replacement therapies (ERTs) and gene therapies for LSDs, including Pompe disease.

A live question and answer session will follow the formal presentations.

About Gregory Enns, MB, ChB
Dr. Gregory Enns is a Professor of Pediatrics in the Division of Medical Genetics at Stanford Medicine and serves as the Director of the Stanford Children's Health Biochemical Genetics Program. He is board-certified in Clinical Genetics and Genomics, as well as Clinical Biochemical Genetics, by the American Board of Medical Genetics and Genomics. Dr. Enns specializes in biochemical genetics, focusing on mitochondrial diseases and lysosomal storage disorders. Dr. Enns is actively involved in clinical trials and has contributed to significant publications in the field of metabolic disorders. His dedication to patient care, research, and education continues to advance the understanding and treatment of genetic metabolic diseases. In addition to his clinical and research roles, Dr. Enns is a member of Stanford's Bio-X institute, which promotes interdisciplinary research in biology and medicine, and the Maternal & Child Health Research Institute (MCHRI). He is also affiliated with the Center for Definitive and Curative Medicine. Dr. Enns earned his MB, ChB from the University of Glasgow in 1990, after completing a Diploma in Medical Science at the University of St. Andrews. He also holds a BA in Biology from Pomona College. His postgraduate training includes a pediatric residency at Children's Hospital Los Angeles and a fellowship in Medical Genetics at UCSF Medical Center.

About Tiffany House
Tiffany House is President of Acid Maltase Deficiency Association (AMDA), established in 1995 to assist in funding research and to promote public awareness of Pompe disease, and Vice-Chair of the International Pompe Association. Tiffany was diagnosed with juvenile-onset Pompe disease at the age of 12, and by age 16, her condition had progressed significantly, requiring her to begin using a wheelchair. Despite being told she would not live past 20, Tiffany has defied expectations through perseverance, medical advancements, and participation in groundbreaking clinical trials. In 1999, Tiffany became the first juvenile patient to participate in clinical trials for enzyme replacement therapy (ERT) in Rotterdam, the Netherlands. She initially received an experimental enzyme derived from transgenic rabbits, which led to gradual improvements in strength and stamina. After a year abroad, she returned to the U.S. for scoliosis surgery at the Mayo Clinic and continued treatment at The University of Texas Health Science Center at San Antonio. In 2002, Tiffany transitioned from the transgenic enzyme to a CHO-derived enzyme due to supply changes. However, this transition, accompanied by a reduction in dosage, led to a decline in her health, highlighting the importance of proper dosing in ERT. Over the years, Tiffany and her family advocated for dosage adjustments, resulting in improvements in her condition. Tiffany’s journey has been marked by resilience, advocacy, and participation in pioneering medical research. Her case has provided invaluable insight into the treatment and management of Pompe disease, influencing ongoing studies and care strategies for patients worldwide.

About M6P Therapeutics
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’ 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’ 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’ mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: www.m6ptherapeutics.com.

Contact us to learn more:
M6P Therapeutics:
314-236-9694
info@m6ptherapeutics.com

Investors:
Bruce Mackle
LifeSci Advisors, LLC
bmackle@lifesciadvisors.com

Legal Disclaimer:

EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.