Apertura Gene Therapy Announces Scientific Founder Presentations on AAV Capsid Engineering Platforms at ASGCT 2023 Annual Meeting

Apertura Gene Therapy, a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today announced that academic collaborator and scientific co-founder, Ben Deverman, PhD, Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, will present at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held in Los Angeles, CA from May 16-20, 2023. Dr. Deverman and members of the Vector Engineering Lab of the Broad Institute will present findings on the founding platforms on which Apertura is based. Both the Fit4Function AAV (adeno-associated virus) capsid engineering platform and a platform approach to engineering AAV capsids targeting known receptors leverage machine learning to accelerate capsid identification and selection.

Members of the Vector Engineering Lab will deliver five oral presentations and two poster presentations on AAV capsid engineering optimization strategies to enhance genetic medicine tropism and delivery efficiency. Dr. Deverman will deliver a presentation called Engineering AAVs to Target and Cross the Brain Vasculature as part of a scientific symposium discussing an array of promising new cell- and gene-based therapeutic approaches for heart and blood vessel diseases.

Details of the presentations are as follows:

Scientific Symposium Details

Title: Engineering AAVs to Target and Cross the Brain Vasculature
Session: New Approaches in Cell and Gene Therapy for Cardiovascular Disease
Presenter: Ben Deverman, PhD
Location: Room 411
Date & Time: Friday, May 19, 9:15-9:40 a.m. PT

Oral Presentation Details

Title: Generative networks create novel receptor targeted AAVs with only 1,200 training examples
Presenting Author: Andrew Barry, PhD
Abstract Number: 43
Session Title: AAV Library Technology
Location: Concourse Hall 150 & 151
Date & Time: Wednesday, May 17, 4:45-5:00 p.m. PT

Title: Development of stabilized AAV capsids for vector engineering
Presenting Author: Simon Pacouret, PhD
Abstract Number: 44
Session Title: AAV Library Technology
Location: Concourse Hall 150 & 151
Date & Time: Wednesday, May 17, 5:00-5:15 p.m. PT

Title: The machine learning-guided Fit4Function platform quantitatively profiles the biodistribution of peptide-modified AAV capsids after a single round of screening in macaque
Presenting Author: Fatma Elzahraa Eid, PhD
Abstract Number: 79
Session Title: AAV Vectors: Clinical and Non-Human Primate Studies
Location: Room 411
Date & Time: Wednesday, May 17, 5:00-5:15 p.m. PT

Title: AAV-mediated CNS-wide gene delivery via a de novo engineered capsid-human receptor Interaction
Presenting Author: Ken Chan, PhD
Abstract Number: 104
Session Title: AAV Engineering for CNS Targeting
Location: West Hall B
Date & Time: Thursday, May 18, 2:00-2:15 p.m. PT

Title: Developing blood-brain barrier-crossing AAV9 variants with reduced sensitivity to neutralizing antibodies
Presenting Author: Simon Pacouret, PhD
Abstract Number: 106
Session Title: AAV Engineering for CNS Targeting
Location: West Hall B
Date & Time: Thursday, May 18, 2:30-2:45 p.m. PT

Poster Presentation Details

Title: Targeting AAV vectors to the CNS via de novo engineered capsid-receptor interactions
Presenting Author: Helen Huang, PhD
Abstract Number: 441
Session Title: Wednesday Poster Session
Date & Time: Wednesday, May 17, 12:00-2:00 p.m. PT

Title: Delivering genes to the brain endothelium to treat lysosomal storage disorder-derived neuropathology
Presenting Author: Nuria Roxana Romero Navarrete
Abstract Number: 908
Session Title: Thursday Poster Session
Date & Time: Thursday, May 18, 12:00-2:00 p.m. PT

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.