Fit4Function AAV capsid engineering platform

	Next higher product group	gene transfer & expression technology
	Status	2022-05-04 existent
	Organisation	Apertura Gene Therapy LLC

Apertura Gene Therapy LLC. (5/4/22). "Press Release: Apertura Announces Fit4Function AAV Capsid Engineering Platform and In Vivo Proof-of-Concept Data to Be Presented at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)". N

Apertura Gene Therapy, a biotechnology company opening opportunities for treating debilitating diseases with limited options for patients, today announced that its academic collaborators will present findings on the Fit4Function AAV capsid engineering platform and non-human primate proof-of-concept data at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held virtually and in Washington, D.C., from May 16–19, 2022.

An oral presentation will discuss Fit4Function, an AAV capsid engineering platform that uses machine learning to guide the selection of capsids with multiple desired traits. Another oral presentation will feature new preclinical data on a novel AAV capsid capable of efficiently crossing the blood-brain-barrier and transducing a majority of neurons in the non-human primate brain. A poster presentation will discuss data related to a second novel AAV capsid capable of transducing key endothelial cells throughout the central nervous system, including arterial, capillary, and venous endothelial cells located in the brain, retina, and spinal cord. Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard and Scientific Founder of Apertura, will also give a pre-meeting workshop on AAV capsid engineering.

Oral Presentation Details:

Title: Fit-4-Function: A Machine Learning-guided Approach for Systematic Multi-trait AAV Capsid Engineering

Presenting Author: Fatma-Elzahraa Eid, Ph.D.
Abstract Number: 1201
Session Title: Novel AAV Capsids for the Brain, Eye and Kidney
Location: Ballroom A
Date & Time: Thursday, May 19, 11:45 a.m. to 12 p.m.

Title: A Promising New Family of Peptide-modified AAV Capsids for Gene Delivery to the Central Nervous System in Non-human Primates

Presenting Author: Ken Chan, Ph.D.
Abstract Number: 1197
Session Title: Novel AAV Capsids for the Brain, Eye and Kidney
Location: Ballroom A
Date & Time: Thursday, May 19, 10:45 a.m. to 11 a.m.

Poster Presentation Details:

Title: A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System
Presenting Author: Trevor Krolak
Abstract Number: 513
Session Title: AAV Vectors — Virology and Vectorology II
Location: Hall D
Date & Time: Tuesday, May 17, 5:30 p.m. – 6:30 p.m.

Pre-meeting Program Presentation:

Session Title: Viral Vector Development Workshop
Presentation Title: AAV Capsid Engineering and Library Approaches
Presenting Author: Ben Deverman, Ph.D.
Location: Room 202
Date & Time: Sunday, May 15, 10:20 a.m. to 10:40 a.m.

About Apertura Gene Therapy

Apertura is a biotechnology company opening opportunities for treating currently intractable diseases. We are uniquely positioned to develop genetic medicines by simultaneously engineering AAV capsids, genetic regulatory elements, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity, and manufacturability. Apertura is committed to growing the field of gene therapy and believes that together we maximize our impact by working with corporate and academic partners, patients, and foundations. Founded on technologies from the Broad Institute and other institutions, and with support from Deerfield Management Company, the company is based at the Cure, Deerfield’s innovation campus in New York City. For more information, please visit our website at www.aperturagtx.com and follow us on LinkedIn and Twitter.

Media Contact: Matthew Corcoran or Nick Chang, MacDougall Advisors, (781) 235-3060, mcorcoran@macdougall.bio or nchang@macdougall.bio