enGene Receives Funding Through Cystic Fibrosis Foundation's Path to a Cure for the Discovery of Novel Gene Therapies to Treat Cystic Fibrosis

BOSTON and MONTRÉAL, Oct. 29, 2020 /PRNewswire/ - enGene Inc., a biotechnology company developing non-viral gene therapies for local administration into mucosal tissues enabled by its proprietary DDX platform, announced today an award from the Cystic Fibrosis Foundation for the discovery of genetic medicines to treat patients with cystic fibrosis (CF).

The award was made as a part of the CF Foundation's $500 million Path to a Cure initiative to accelerate the discovery and development of treatments that address the underlying cause of the disease.

Affecting over 75,000 patients worldwide, CF is a genetic disease caused by mutations in a gene known as the cystic fibrosis transmembrane conductance regulator (CFTR) that render a non-functional CFTR protein. Consequently, multiple organs are affected by disease, chief among them the lungs, where chronic infections and a worsening ability to breathe leads to progressive lung damage and premature death. Patients with nonsense and other rare mutations in both copies of the CFTR gene currently have no therapies that treat the underlying cause of the disease.

"Gene therapy holds promise for the treatment of CF by delivering a functional copy of the CFTR gene to the lungs to restore function and alleviate disease. enGene is developing a DDX-based inhalable formulation to carry DNA to the airways with the goal of functional complementation of CFTR mutations. We are thrilled to have the support of the Cystic Fibrosis Foundation to discover novel gene therapy candidates for patients with CF," commented Jose Lora, CSO of enGene.

In developing an inhalable gene therapy for CF, enGene is coupling a non-viral DNA payload to its biocompatible DDX carrier in an effort to create genetic medicines that allow repeatable and titratable dosing to achieve meaningful efficacy.

"Gene therapies have made a remarkable impact in many fields of medicine, but unlocking their full potential in mucosal tissues such as the lung has been elusive, leaving many patients with CF without available treatment options. We are honored to be working with the CF Foundation to accelerate our research and development efforts towards improving and extending the lives of all CF patients," said Jason Hanson, enGene's President and CEO.

About enGene Inc.
enGene Inc. is a biotechnology company developing a proprietary non-viral gene therapy platform for localized delivery of nucleic acid payloads to mucosal tissues. The dually derived chitosan (DDX) platform has a high-degree of payload flexibility including DNA and various forms of RNA with broad tissue and disease applications. In addition to developing gene therapies for the lungs, enGene has developed a unique dual-immune activator for patients with non-muscle invasive bladder cancer which has completed IND-enabling studies. The company is evolving its technology to enable applications in multiple mucosal tissues with areas of high unmet medical need. www.engene.com/

Note regarding forward-looking statements
This press release contains certain "forward-looking statements" that reflect the Company's beliefs and assumptions based on currently available data and information. These forward-looking statements fall within the meaning of the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "target," "believe," "expect," "will," "may," "anticipate," "estimate," "would," "positioned," "future," and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on enGene's current beliefs, expectations, and assumptions that by definition involve risks, uncertainties, that are difficult to predict and are subject to factors outside of management's control and that could cause actual results to differ substantially from statements made including but not limited to: risks associated with the success of preclinical studies, clinical trials, research and development programs, as well as regulatory approval processes. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. enGene has no approved drugs available for sale marketing at this time and may never have an approved drug. You are cautioned not to rely on enGene's forward looking statements, which are only made as of the date hereof. The Company is under no obligation to update these statements.

SOURCE enGene