DOK7

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. SOURCE Amplo Biotechnology WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SAN DIEGO, May 29, 2023 /PRNewswire/ -- Amplo Biotechnology, a leading US-based biotech company dedicated to developing innovative AAV-based gene therapies for neuromuscular junction disorders announced the award of a Fast Track Phase I/II STTR grant from the NIH-NIAMS. This highly competitive grant will provide substantial funding for the advancement of AMP-201, an AAV-ColQ gene therapy designed to address the severe congenital myasthenic syndrome (CMS) caused by Collagen Q (ColQ) Deficiency. Under an exclusive licensing agreement, Amplo Biotechnology has gained access to AAV-ColQ data developed by Professor Kinji Ohno's laboratory at Nagoya University, which forms the basis of AMP-201. In the Fast Track STTR project, Amplo will collaborate with Professor Ricardo Maselli, a CMS expert neurologist and researcher at UC-Davis. Amplo Biotechnology's development of AMP-201 builds upon it's progression of AMP-101, an AAV-DOK7 gene therapy targeting Dok-7 congenital myasthenic syndrome, which Amplo expects to bring to clinical trial in 2024. Upon demonstrating initial safety and efficacy, the company plans to expand into other neuromuscular conditions, leveraging the vast potential of AMP-101 and AMP-201 to significantly enhance the quality of life for the majority of CMS patients. "Amplo is thrilled to collaborate with UC-Davis in the development of AMP-201, addressing the critical unmet needs of patients suffering from ColQ CMS. By combining the power of Amp-101 and AMP-201, we are confident in our ability to revolutionize the lives of CMS patients," expressed Dr. Patricio Sepulveda, CEO of Amplo Biotechnology, who holds a Ph.D. in the field and an MBA. About Amplo Biotechnology: Amplo Biotechnology is a leading late-stage preclinical AAV gene therapy company, committed to leveraging gene therapy advancements to improve neuromuscular junction function across various medical conditions. The company's flagship program, AMP-101, has successfully undergone rigorous preclinical studies, enabling the submission of a Clinical Trial Application in the UK for Dok7 CMS. Amplo Biotechnology's technologies are rooted in ground-breaking research conducted at renowned institutions such as the University of Tokyo, Oxford University, and Nagoya University. For media inquiries and further information, please contact: Patricio Sepulveda [email protected] SOURCE Amplo Biotechnology

NEW YORK, Oct. 6, 2021 /PRNewswire/ -- Amplo Biotechnology, a privately held US-based biotech focused on developing AAV (Adeno-associated Virus)-based gene therapies for diseases affecting the neuromuscular junction, today announced the successful closing of an undisclosed Series Seed financing. The Seed round was led by BioBrit, LLC., joined by Casdin Capital, SeedFolio and small group of sophisticated, biotech angel investors. In conjunction with the financing, Dan Bradbury (BioBrit, LLC.) has been appointed Chair of the Board. It was also announced that Amplo was awarded a NIH Fast-Track Phase I/II SBIR grant for the development of AMP-101 gene therapy. The latest funding round will be used to advance AMP-101, an AAV carrying Dok-7, to large scale manufacturing and to complete IND-enabling studies. AMP-101 was invented as a means to treat Dok-7 congenital myasthenic syndrome. AMP-101 has also shown to revert age-related loss in muscle function by improving the neuromuscular junction and decreasing denervation in mice. AMP-101 has also shown to increase survival and functional benefits in mouse models of Amyotrophic Lateral Sclerosis and Emery-Dreyfuss muscular dystrophy. Amplo secured FDA orphan drug designation for AMP-101 in January 2020. Amplo, plans to begin clinical trials in Dok-7 congenital myasthenic syndrome in 2023, with aims to move into other neuromuscular conditions once initial safety and efficacy are demonstrated. "When we think about AMP-101, we see the potential to treat many neuromuscular diseases with a single gene therapy. The closing of the Seed financing will allow us to rapidly execute on a plan that has received FDA and MHRA input," said Patricio Sepulveda Ph.D. MBA, CEO of Amplo Biotechnology. In addition, AMP-101, Amplo is developing other AAVs to treat diseases of the neuromuscular junction. About Amplo Biotechnology Amplo is a late preclinical AAV gene therapy company focused on developing therapies for congenital myasthenic syndromes and conditions of the neuromuscular junction.