Homology Medicines Announces Presentations at Upcoming American Society for Gene & Cell Therapy Annual Meeting

BEDFORD, Mass., April 29, 2020 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today upcoming presentations demonstrating the broad applicability of its in vivo gene therapy and nuclease-free gene editing platform across multiple disease areas. Presentations will also include mechanistic data further characterizing Homology’s novel AAVHSC vectors and details on the commercial manufacturing process and internal GMP production capabilities at Homology. These data will be presented during the virtual American Society for Gene & Cell Therapy (ASGCT) 23rd Annual Meeting in the online poster sessions May 12 - 14, 2020.

“We are looking forward to sharing data that demonstrate the potential of our AAVHSC genetic medicines platform to treat rare genetic diseases such as PKU and MLD,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our presentations represent how we continue to advance our understanding of Homology’s proprietary AAVHSC vectors through characterization of cellular transduction, impact on cell cycle, and biodistribution to disease-relevant cell types after a single I.V. administration. Homology’s leadership in gene therapy and gene editing manufacturing will be the subject of presentations highlighting our efficient commercial process and scalable internal GMP manufacturing platform, which currently serves the needs of our clinical and preclinical programs. Additionally, our contributions to the field of gene editing will be featured in Homology data detailing molecular methods used to measure homologous recombination-based genomic integration, methods that we believe are important to characterize changes to the genome, measure integration efficiency and compare results across studies and platforms.”

Homology’s ASGCT 2020 presentations include:

In Vivo, Nuclease-Free Gene Editing for PKU
Molecular Characterization of Precise In Vivo Targeted Gene Editing in Human Cells
using AAVHSC15, a New AAV Derived from Hematopoietic Stem Cells (AAVHSC)

  • Tuesday, May 12; 5:30 - 6:30 p.m.
  • Abstract #: 227

Scalable Manufacturing
Molecular Design and Characterization of Packaging Plasmid Sequences for
Improved Production of Novel Clade F AAVHSCs

  • Tuesday, May 12; 5:30 - 6:30 p.m.
  • Abstract #: 445

Development and Scalability of Transfection-Based Production and Purification of
Novel Clade F Adeno-Associated Viruses Isolated from Human Hematopoietic Stem Cells (AAVHSCs)

  • Thursday, May 14; 5:30 - 6:30 p.m.
  • Abstract #: 1248

In Vivo Gene Therapy for MLD
Gene Therapy for Metachromatic Leukodystrophy (MLD) That Crosses the Blood-Nerve and
Blood-Brain Barriers in Mice and Non-Human Primates

  • Wednesday, May 13; 5:30 - 6:30 p.m.
  • Abstract #: 590

In Vivo Transduction of Hematopoietic Stem Cells
In Vivo Transduction of Murine Hematopoietic Stem Cells after Intravenous Injection of
AAVHSC15 and AAVHSC17

  • Wednesday, May 13; 5:30 - 6:30 p.m.
  • Abstract #: 600

AAVHSC Platform
Role of Terminal Galactose in Cellular Uptake, Intracellular Trafficking, and Tissue Tropism
Using Adeno-Associated Viruses Isolated from Human Stem Cells (AAVHSCs)

  • Wednesday, May 13; 5:30 - 6:30 p.m.
  • Abstract #: 570

AAVHSCs Transduction Does Not Significantly Elicit p53-Mediated Apoptosis or Alter Cell Cycle in Human iPSCs and Primary Cells When Compared to Non-Clade F AAV Vectors

  • Thursday, May 14; 5:30 - 6:30 p.m.
  • Abstract #: 1011

The abstracts are available on the ASGCT website.

About Homology Medicines, Inc.

Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of preclinical data; our beliefs regarding our manufacturing capabilities; beliefs about preclinical data; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; the impact of the COVID-19 pandemic on our operations, the continuity of our business, including our preclinical studies and clinical trials, and general economic conditions; and significant costs as a result of operating as a public company. These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2019 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Company Contacts:
Theresa McNeely
Chief Communications Officer and
Patient Advocate
tmcneely@homologymedicines.com
781-301-7277

Media Contact:
Cara Mayfield
Senior Director, Patient Advocacy
and Corporate Communications
cmayfield@homologymedicines.com
781-691-3510