Translate Bio Highlights mRNA Platform Potential for the Treatment of Cystic Fibrosis (CF) at the 34th Annual North American Cystic Fibrosis Conference
-- Lead candidate MRT5005 leverages Translate Bio’s proprietary LNP and mRNA platform and is the only clinical-stage mRNA therapeutic in development for the treatment of CF --
-- Second CF program, next-generation CFTR mRNA, applies advances in mRNA science, LNP chemistry and CFTR biology --
-- mRNA capabilities continue to expand to address unmet needs in CF and other pulmonary diseases --
In an oral presentation entitled, “Delivering CFTR mRNA: An Inhaled, Mutation-agnostic Approach to Treat CF,” the Company discusses the potential of nucleic acid therapies to treat the underlying cause of cystic fibrosis (CF), particularly through the promise of messenger RNA (mRNA) therapeutics. The presentation underscores the remaining unmet need in treating CF and provides an overview of the development of its lead clinical candidate, MRT5005, currently being evaluated in a Phase 1/2 clinical trial in people with CF.
In addition, the oral presentation and a poster presentation entitled, “Design of Next Generation CFTR mRNA Therapeutics (MRT) for the Treatment of Patients with Cystic Fibrosis,” summarize preclinical research related to the design of the Company’s next-generation CF program. Both presentations describe advancements in the Company’s mRNA technology as applied to the treatment of CF, including ideal target profile and preclinical data demonstrating that the design of novel mRNA sequences encoding rationally engineered CFTR protein produced highly active ion channels and enhanced protein expression when compared to a reference sequence encoding the native protein in vitro. The presentations also highlight the potential benefits of a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) mRNA which may include lower dosing requirements and/or reduced administration time. Preclinical studies are ongoing to select the optimal mRNA sequence and lipid nanoparticle (LNP) for Translate Bio’s next generation CFTR mRNA product.
“We are steadfast in our commitment to the CF community, especially those without any efficient, corrective treatment, and we believe that mRNA therapeutics have the potential to benefit all people with CF, regardless of underlying genetic mutation,” said
The NACFC presentations highlight multiple innovations across the Company’s mRNA therapeutic platform (MRTTM) involved in advancing a next-generation CFTR program including:
- Optimization of mRNA sequences and improvements in the amount of protein produced per unit of mRNA delivered;
- Deep evaluation of rationally engineered CFTR proteins with potential to increase the activity of the CFTR ion channel; and
- Incorporation of novel proprietary lipids for inhaled delivery.
|Title:||S02.4: Delivering CFTR mRNA: An Inhaled, Mutation-agnostic Approach to Treat CF|
|Session:||S02--PTC-APP&D-NT: Novel Nucleic Acid Strategies to Treat the Fundamental CF Defect|
|Date and time:
|Title:||Design of Next Generation CFTR mRNA Therapeutics (MRT) for the Treatment of Patients with Cystic Fibrosis|
The NACFC portal can be accessed here. Links to the poster and oral presentation will be available on Translate Bio’s investor website on
MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein. The
About the MRT5005 Phase 1/2 Clinical Trial
The randomized, double-blind, placebo-controlled Phase 1/2 clinical trial of MRT5005 is designed to enroll at least 40 adult patients with CF who have two Class I and/or Class II mutations. The primary endpoint of the trial will be the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization. Percent predicted forced expiratory volume in one second (ppFEV1), which is a well-defined and accepted endpoint measuring lung function, will also be measured at pre-defined timepoints throughout the trial. The Phase 1/2 clinical trial of MRT5005 for the treatment of CF is being conducted in collaboration with the Cystic Fibrosis Foundation Therapeutics Development Network and the Emily’s Entourage Patient Registry. In
About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for MRT5005 to address the underlying cause of CF and benefit all patients, including the potential benefits of nucleic acid therapies and of a next-generation CFTR mRNA therapeutic; Translate Bio’s plans to advance MRT5005 and its next-generation CFTR program; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: the current and potential future impacts of the COVID-19 pandemic on the Company’s business, financial condition, operations and liquidity; Translate Bio’s ability to advance the development of its platform and programs under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; the successful advancement of the collaboration agreement between
Source: Translate Bio, Inc.