Biotech Start-Up to Discover Cystic Fibrosis Therapy

Lung x-ray image (Toubibe, Pixabay. https://pixabay.com/photos/x-ray-image-roentgen-thorax-568241/

19 June 2023. A new biotechnology company is advancing research to find a comprehensive treatment for cystic fibrosis, and staked to a €2 million seed investment from its founder. Anoat Therapeutics in Paris is formed recently by life science venture investor AdBio partners, also in Paris, from a fund started last year with Inserm Transfert, the technology transfer arm of Inserm, the French National Institute of Health and Medical Research.

Cystic fibrosis is a progressive inherited disorder affecting the lungs, pancreas, and other organs. The disease results from various mutations in the cystic fibrosis transmembrane conductance regulator or CFTR gene and malfunctioning protein that keeps the element chloride away from cell surfaces. Chloride attracts water, and without more water, substances like mucous in the lungs thicken and become sticky, clogging the airways and trapping infectious bacteria. As a result, people with cystic fibrosis — estimated at 105,000 worldwide — are more susceptible to infections, respiratory failure, and inflammation.

Up to now, says Anoat Therapeutics, most cystic fibrosis treatments addressed specific CFTR gene mutations. Yet, in a paper published earlier this month, a team from Armand-Trousseau pediatric hospital affiliated with the Sorbonne in Paris and Inserm says those approaches leave out other people with cystic fibrosis resulting from other more random CFTR mutations. The researchers say a more comprehensive approach to cystic fibrosis can address all CFTR defects, with an antisense oligonucleotide, a short synthetic nucleic acid component that activates an alternative signaling channel. In this case, that channel called TMEM16A, compensates for a defective CFTR. In a preclinical study with cell lines and lab mice, the antisense oligonucleotide enhances mucous clearance and chloride activity, plus extends the lifespan of mice with little sign of toxicity.

First company formed from partnership

AdBio partners formed Anoat Therapeutics a short time ago — the company has yet to post its own web site — to advance this research through drug discovery and preclinical stages, and identify a treatment candidate. “Based on highly promising proof-of-concept data and relying on a network of physicians and patient associations,” says Anoat CEO Vincent Bischoff in an AdBio partners statement, “the Anoat team will strive to meet the medical needs of CF patients who, to date, remain without any treatment. We aim to provide a therapeutic option that will help alleviate the burden of their life-threatening condition.”

Anoat Therapeutics is the first company formed from a partnership begun in May 2022 between Inserm Transfert, Inserm’s technology transfer division, and AdBio partners, with Inserm Transfert taking part in AdBio’s second investment fund called AFB Fund 2. The collaboration and the €86 million fund, say the partners, are designed to help new life science businesses through their early stages and advance the entrepreneurial skills of scientific founders. AFB Fund 2 is providing Anoat with €2 million (US$ 2.19 million) in seed funds.

Pascale Augé, board chair at Inserm Transfert, notes that the founding of Anoat Therapeutics “shows that our respective fields of expertise complement each other and that a close relationship between Inserm Transfert and AdBio Partners facilitates the emergence and early financing of innovative life sciences companies.”

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