Investors & Media

Multiple Poster Presentations Highlight Versatility and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell Therapy

LONDON and NEW YORK, Oct. 04, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company will exhibit 15 poster presentations at the European Society of Gene and Cell Therapy (ESGCT) 2022 Annual Congress, which will be held from October 11-14, 2022, in Edinburgh, Scotland.

The posters will include data from MeiraGTx’s novel gene regulation platform, including the first data demonstrating the potential to regulate CAR-T, as well as data from the Company’s promoter platforms and several new, optimized pre-clinical programs addressing severe unmet needs for indications such as amyotrophic lateral sclerosis (ALS) and Wilson’s disease. In addition, the Company will have presentations on its proprietary viral vector manufacturing technology and potency assay development.

“We’re pleased to present data illustrating the depth and versatility of MeiraGTx’s scientific platforms,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “The 15 published abstracts at this year’s ESGCT Congress reflect the extraordinary productivity of our research efforts in developing new technologies and applying them to the design of optimized genetic medicines, as well as innovation in manufacturing and process development technology. I am particularly excited for us to present our riboswitch gene regulation technology applied to cell therapy for the first time, in this case the regulation of CAR-Ts, which is a huge area of scientific and clinical interest,” continued Dr. Forbes. “We look forward to presenting these data highlighting our innovative platform technologies and broad R&D capabilities.”

Abstract Title (P101): AI-driven promoter optimization at MeiraGTx
Session Title: Advances in viral and non-viral vector design
Date: October 12, 2022

Abstract Title (P124): Promoter Engineering Platform at MeiraGTx
Session Title: Advances in viral and non-viral vector design
Date: October 13, 2022

Abstract Title (P243): UPF1 delivered by novel expression-enhanced promoters protects cultured neurons in a genetic ALS model
Session Title: CNS and sensory
Date: October 12, 2022

Abstract Title (P254): Optimization and scale-up of AAV2-AQP1 production using a novel transient transfection agent
Session Title: Developments in manufacturing and scale up
Date: October 13, 2022

Abstract Title (P264): Designing and screening formulations to improve manufacturability and distribution of AAV gene therapies
Session Title: Developments in manufacturing and scale up
Date: October 13, 2022

Abstract Title (P270): Use of anion exchange chromatography to provide high empty AAV capsid removal and product yields
Session Title: Developments in manufacturing and scale up
Date: October 13, 2022

Abstract Title (P320): Multivariate analysis for increased understanding of MeiraGTx upstream process
Session Title: Developments in manufacturing and scale up
Date: October 13, 2022

Abstract Title (P362): Development of AAV-UPF1 gene therapy to rescue ALS pathophysiology using microfluidic platforms
Session Title: Disease models (iPS derived and organoids)
Date: October 13, 2022

Abstract Title (P399): Titratable and reversible control of CAR-T cell receptor and activity by riboswitch via oral small molecule
Session Title: Engineered T and NK CARs and beyond
Date: October 12, 2022

Abstract Title (P436): Novel riboswitches regulate AAV-delivered transgene expression in mammals via oral small molecule inducers
Session Title: Gene and epigenetic editing
Date: October 13, 2022

Abstract Title (P553): Development of optimized ATP7B gene therapy vectors for the treatment of Wilson’s Disease with increased potency
Session Title: Metabolic diseases
Date: October 12, 2022

Abstract Title (P554): A CNS-targeted gene therapy for the treatment of obesity
Session Title: Metabolic diseases
Date: October 13, 2022

Abstract Title (561): Riboswitch-controlled delivery of therapeutic hormones for gene therapy
Session Title: Metabolic diseases
Date: October 12, 2022

Abstract Title (P622): Riboswitch-controlled delivery of therapeutic antibodies for gene therapy
Session Title: Other
Date: October 13, 2022

Abstract Title (P630): Improving AAV in vitro transducibility for cell-based potency assay development
Session Title: Other
Date: October 13, 2022

About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, and a transformative gene regulation platform technology which allows tight, dose responsive control of gene expression by oral small molecules with dynamic range that can exceed 5000-fold. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring, and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: ocular, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and severe forms of xerostomia. Though initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx plans to expand its focus to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visit www.meiragtx.com.

Forward Looking Statement
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 product candidate development and our pre-clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause 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, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov. These and other important factors 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, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:
MeiraGTx
Investors@meiragtx.com

Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com