Rare Daily Staff

Eloxx Pharmaceuticals and Metagenomi, in separate announcements, said the Cystic Fibrosis Foundation has awarded them funding to further develop their treatments for cystic fibrosis patients.

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. In cystic fibrosis patients, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional.

Eloxx received an award of up to $2.6 million to identify optimized oral ribosome modulating agents (RMAs) for further development in the treatment of CF patients with nonsense mutations.

The company is engaged in the science of ribosome modulation, leveraging both its proprietary chemistry technology platform in an effort to develop novel RMAs and its library of Eukaryotic Ribosome Selective Glycosides. Eloxx’s lead experimental product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins.

“The development of a Ribosome Modulating Agent for the treatment of CF with support from the Cystic Fibrosis Foundation complements our ongoing ELX-02 program, currently in phase 2 clinical trials,” said Sumit Aggarwal, president and CEO of Eloxx. “Data from these phase 2 trials is expected in the second half of this year.”

Gene editing biotech Metagenomi also received an award from the Cystic Fibrosis Foundation to identify and evaluate novel gene editing systems for the development of therapeutics to treat cystic fibrosis.

“Cystic fibrosis is a complex, chronic genetic disease and there remains a significant unmet need for new therapies to help patients,” said Brian Thomas, co-founder and CEO of Metagenomi. “Metagenomi’s gene editing capabilities are transformational and could provide opportunities for gene editing-based therapeutics that are not possible with other systems.”

According to the terms of the award, Metagenomi will work to leverage its proprietary metagenomics database to screen and characterize novel gene editing systems with high on-target specificity that are suitable for in vivo gene editing at therapeutically relevant sites in the CFTR gene. Other terms of the award have not been disclosed.

Photo: Brian Thomas, co-founder and CEO of Metagenomi

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