Zikani Therapeutics has raised $7.5 million in Series A funding to advance work, using its TURBO-ZM technology platform, into potential therapies for rare disorders caused by nonsense mutations, including cystic fibrosis (CF).
TURBO-ZM is a proprietary platform that enables Zikani to rapidly produce ribosome modulating agents (RMAs). These compounds work by targeting ribosomes — small structures responsible for the production of proteins from a template RNA sequence — and for modulating their activity in a disease-specific fashion.
Zikani is planning to use the new funds to develop RMAs specifically designed to overcome the effects of nonsense mutations, in which an alteration in a single nucleotide of the gene’s DNA sequence leads to the introduction of a stop codon, and the production of a shorter and non-functional protein.
Nucleotides are the building blocks of DNA, and codons are sequences of three nucleotides that encode one amino acid (the building blocks of proteins).
The idea is to generate disease-specific RMAs that allow ribosomes to ‘ignore’ the stop codons introduced by nonsense mutations. In other words, to generate compounds that promote ribosomes’ ability to read-through the entire sequence, resulting in the production of a working, normal-size protein.
In the particular case of CF, this technology platform could allow the production of a functional CFTR protein (the defective protein in patients with the disease).
The company is working on developing RMAs for rare diseases caused by nonsense mutations, including class 1 CF, familial adenomatous polyposis, APC mutant colon cancer, and recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB). It reports promising pre-clinical data has been obtained for these diseases.
“We’re highly encouraged by the data we’ve generated to date and are working rapidly to advance compounds into pre-clinical development. This financing … is a vote of confidence for our promising technology platform,” Sumit Aggarwal, president and CEO of Zikani Therapeutics, said in a press release.
“As we look ahead, our focus is to maximize the funding and take the steps necessary to generate greater recognition for RMAs and form collaborations with partners that will benefit from our scientific platform,” Aggarwal added.
“We’re pleased to support Zikani as an emerging company that is using the strength of its science and a refined strategy to develop novel ribosome modulators for rare diseases caused by nonsense mutations,” said Raj Parekh, a general partner of Advent Life Sciences.
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