SAN DIEGO, Oct. 17, 2024 /PRNewswire/ -- Amplo Biotechnology, a biotechnology company developing genetic medicines for the treatment of neuromuscular diseases, announced the presentation of three posters at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, taking place from October 22-25, 2024, in Rome, Italy. Amplo will feature three poster presentations detailing its platform approach to developing programmatic gene therapies, new preclinical data for Dok-7 gene therapy (AMP-101), and lead candidate activities for its novel Collagen Q therapy (AMP-201).
"We are pleased to present data at ESGCT demonstrating the potential of our lean drug development strategy that supports the commercial viability of gene therapies in rare- and super-rare diseases by leveraging the ecosystem's knowledge," said Patricio Sepulveda, PhD MBA, Chief Executive Officer at Amplo Biotechnology.
Details for the poster presentations:
Abstract Title: Development of a platform gene therapy approach for Congenital Myasthenic Syndromes
Presenting Author: Patricio V. Sepulveda S., PhD MBA
Date: Tuesday, October 22, 2024
Abstract Title: Pre-clinical development of gene augmentation therapy to restore Acetylcholinesterase activity in Collagen Q Congenital Myasthenic Syndrome using a platform approach to AAV gene therapy development.
Presenting Author: Claudia Canzonetta, PhD
Date: Tuesday, October 22, 2024
Abstract Title: The pre-clinical development of gene augmentation therapy to stabilize the neuromuscular junction in Dok-7 Congenital Myasthenic Syndrome reveals a mechanism of action that has the potential to treat many neuromuscular junction diseases using the same vector.
Presenting Author: Paul Al Hawkins
Date: Wednesday, October 23, 2024
About Amplo Biotechnology
Amplo is a late, pre-clinical genetic medicines company focused on therapies for rare and super-rare diseases.