- Homology's New GMP Manufacturing Facility Preparing for Production at 500 Liter Scale -
- Orphan Drug Designation Granted for HMI-102 PKU Gene Therapy in the U.S. and E.U. -
BEDFORD, Mass., Jan. 07, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced that it achieved all corporate goals for 2018, positioning the Company to start a Phase 1/2 clinical trial of the Company's first gene therapy program in adults with phenylketonuria (PKU), with initial data expected in 2019. Homology closed out the year by nominating development candidates for two additional programs focused on pediatric rare genetic diseases, both of which have entered into IND-enabling studies. These candidates comprise a nuclease-free in vivo gene editing approach for PKU and a gene therapy approach for metachromatic leukodystrophy (MLD), Homology's lead central nervous system (CNS) program and a fatal pediatric lysosomal storage disorder. Pipeline advancements are expected to be supported by Homology's newly completed GMP gene therapy and gene editing manufacturing facility.
"We entered 2018 with aggressive goals to translate our technology into development programs that have the potential to cure people living with rare genetic diseases," said Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. "Through the hard work of our team and the rapid progression of our dual gene therapy and gene editing platform, we are pleased that we met all of our 2018 goals and enter 2019 with strong momentum to move our pipeline forward."
Dr. Tzianabos continued, "We begin this year on track to start and report initial data from our Phase 1/2 gene therapy trial for adults with PKU, and we have begun IND-enabling studies with our first gene editing program for children with PKU. Our approach with PKU exemplifies the duality and flexibility of our technology platform where we choose the best genetic medicines approach based on human biology and the underlying cause of disease. Since our naturally derived AAVs cross the blood-brain-barrier, we are also targeting diseases of the central and peripheral nervous system, beginning with our lead CNS gene therapy program for MLD. With two additional programs now in IND-enabling studies, we expect that our new GMP manufacturing facility will provide us with control over the pace of development for these and future pipeline programs."
Key 2018 Accomplishments and 2019 Priorities
HMI-102 In Vivo Gene Therapy for Adults with PKU
HMI-103 In Vivo Gene Editing Treatment for Pediatric Patients with PKU
HMI-202 In Vivo Gene Therapy Treatment for Lead CNS Program, MLD
Dual Technology Platform
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 .
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 statements regarding upcoming events and presentations; advancing our novel gene therapy and gene editing technology platform and pipeline; our expectations surrounding initiation of clinical trials for our PKU gene therapy program, release of clinical data, and timing thereof; the progress of IND-enabling studies for our PKU gene editing and MLD gene therapy programs; our beliefs regarding our manufacturing capabilities and ability to accommodate both gene therapy and gene editing pipeline programs; our goal of improving the lives of patients with rare genetic diseases; beliefs about our position as a leader in the development of genetic medicines; and the sufficiency of our cash position. 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: the fact that 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 marketable products; the early stage of our development efforts; our failure or the failure of our collaborators to successfully develop and commercialize drug candidates; potential unforeseen events during clinical trials could cause delays or other adverse consequences; 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; the inability to obtain orphan drug exclusivity; failure to obtain international marketing approval; failure to obtain U.S. marketing approval; ongoing regulatory obligations; effects of significant competition; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; the price of our common stock may be volatile; significant costs as a result of operating as a public company; and any securities class action litigation. These and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2018 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.
|Investor Contact:||Media Contact:|
|Theresa McNeely||Cara Mayfield|
|SVP, Corporate Communications||Director, Corporate Communications|
|& Patient Advocacy