Homology Medicines Announces Four Presentations on its Gene Editing and Gene Therapy Platform at the American Society of Gene & Cell Therapy Meeting

BEDFORD, Mass., April 24, 2017 – Homology Medicines, Inc., a genetic medicines company translating proprietary gene editing and gene therapy technologies into novel treatments for patients, announced today that data from its gene editing and gene therapy platform will be presented in an oral presentation and three poster presentations during the American Society of Gene & Cell Therapy (ASGCT) 20th Annual Meeting at the Marriott Wardman Park Hotel in Washington, D.C. from May 10-13, 2017.

During the oral presentation, preclinical data will be presented for the first time demonstrating the precise, on-target in vivo gene editing capabilities of Homology Medicines’ proprietary adeno-associated virus hematopoietic stem cell (AAVHSC) vectors. In poster presentations, the Company will also present additional new data related to its AAVHSC vectors, including quantitative gene editing data, biodistribution data in the peripheral and central nervous systems and data characterizing the frequency of pre-existing neutralizing antibodies in humans.

“Our presentations at ASGCT highlight the significant therapeutic potential of our gene editing and gene therapy platform, including its unique capabilities to induce precise and efficient gene editing without the need for nucleases as well as the superior biodistribution of our AAVHSC vectors for gene therapy,” said Arthur Tzianabos, Ph.D., Chief Executive Officer of Homology Medicines. “We look forward to building upon this scientific information as we advance our platform to develop potential cures for patients with rare genetic diseases.”

Homology Medicines’ presentations at the ASGCT Meeting are outlined below.

Oral Presentation

Title: AAVHSC Vectors Mediate Highly Precise and Efficient Homologous Recombination-Based Gene Editing
Date, Time: May 13, 2017, 10:30 a.m. ET
Room: Marriott Salon 2
Abstract Number: 746

Poster Presentations

Title: Evaluation of On-Target and Off-Target Precision of AAVHSC-Mediated Genome Editing
Date, Time: May 10, 2017, 5:30 p.m. ET
Room: Exhibit Hall A & B South
Abstract Number: 172

Title: Prevalence of Neutralizing Antibodies Targeting Two Novel Clade F AAV in Human Sera
Date, Time: May 10, 2017, 5:30 p.m. ET
Room: Exhibit Hall A & B South
Abstract Number: 187

Title: Widespread Transduction of the Central Nervous System Following Systemic Delivery of AAVHSC17 in Non-Human Primates
Date, Time: May 12, 2017, 5:45 p.m. ET
Room: Exhibit Hall A & B South
Abstract Number: 568

Abstracts are available on the ASGCT Meeting website.

About Homology Medicines, Inc.

Homology Medicines is a genetic medicines company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. The combination of a new multidimensional technology platform and a management team that has successfully developed and commercialized rare disease therapies uniquely positions the Company to move beyond the current limitations of gene therapy and gene editing approaches to improve patient care. Homology Medicines has built foundational intellectual property on gene editing and gene therapy using vectors derived from naturally occurring human adeno-associated viruses (AAVs). The Company’s technology is precise, on-target and highly efficient for in vivo editing of genetic mutations. The unique team and technology create a significant opportunity for Homology Medicines to rapidly advance a diverse pipeline of new medicines that address and potentially cure the underlying cause of genetic diseases. For more information, please visit www.homologymedicines.com.

Cara Mayfield
Director, Corporate Communications
cmayfield@homologymedicines.com
781-301-7277, ext. 141

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