Amplo Biotechnology Announces Three Presentations at the European Society of Gene and Cell Therapy 31st Annual Congress

Details for the poster presentations: Details for the poster presentations: Abstract Title:  Development of a platform gene therapy approach for Congenital Myasthenic SyndromesPresenting Author:  Patricio V. Sepulveda S., PhD MBADate:  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...

SAN DIEGO, 17/10/2024 (informazione.it - comunicati stampa - salute e benessere)

 Development of a platform gene therapy approach for Congenital Myasthenic Syndromes
 Patricio V. Sepulveda S., PhD MBA
 Tuesday, October 22, 2024 

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.
 Claudia Canzonetta, PhD
 Tuesday, October 22, 2024 

 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.
 Paul Al Hawkins
 Wednesday, October 23, 2024

Amplo is a late, pre-clinical genetic medicines company focused on therapies for rare and super-rare diseases.

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