In light of this, the company is discontinuing the development of its lead candidate MRT5201 for ornithine transcarbamylase deficiency — a metabolic liver enzyme disorder — and will prioritize and expand the development of its pulmonary disease programs, according to a press release.
This decision was based on positive results from an ongoing Phase 1/2 trial (NCT03375047), called RESTORE-CF, exploring the safety and effectiveness of MRT5005 candidate in patients with cystic fibrosis. In contrast, preclinical studies have failed to demonstrate desired therapeutic features for MRT5201, which supported its discontinuation.
“We believe that the success to date in our cystic fibrosis program positions us well to build on our lung delivery platform, and maximize the potential of our mRNA technology in additional pulmonary diseases with unmet medical need,” said Ronald Renaud, CEO of Translate Bio.
Translate Bio develops new mRNA therapies that aim to restore or enhance the activity of targeted proteins. It engineers mRNA molecules that will provide the necessary information to cells to produce the desired protein. These mRNA molecules are incorporated into tiny fatty vesicles (nanoparticles) that work as vehicles to deliver the mRNA to cells.
Given the company’s lipid (fatty) nanoparticle delivery system, it is possible to transport and deliver mRNA therapies to a broad range of cells in the body.
“We are always evaluating various methods for the delivery of mRNA and we continue to believe that LNPs [lipid nanoparticles] are currently the most promising technology with advantages over other approaches,” Renaud said. “We design each LNP with distinct chemical structures and formulations specific to the target organ and route of administration in order to optimize safety and potency.”
MRT5005, for example, was designed using the company’s proprietary mRNA therapeutic platform to normalize the function of the CFTR protein, which is impaired in patients with cystic fibrosis due to genetic mutations. MRT5005 is encapsulated in lipid nanoparticles that are nebulized to reach the lungs.
The most recent results from the RESTORE-CF trial demonstrated that treatment with MRT5005 could effectively improve respiratory function, while showing a positive safety and tolerability profile.
Translate Bio has now announced that it is conducting preclinical studies focused on identifying therapeutic candidates for the treatment of pulmonary arterial hypertension, primary ciliary dyskinesia, and idiopathic pulmonary fibrosis.
“We are excited about the robust mRNA delivery effort underway at Translate Bio, and we’ll continue to apply our expertise in the development of novel lipids,” Renaud concluded.
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