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Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the ...
Anne Douar
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Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis

Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis

 Funding from French Government as part of the France Health Innovation Plan 2030

Vivet Therapeutics, a French clinical stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic disorders, announces the financing of €4.9 million received from the French government to advance the development of a gene therapy for the treatment of cerebrotendinous xanthomatosis (CTX). This rare autosomal recessive genetic disorder affects the body’s ability to metabolize cholesterol. Patients with CTX are unable to break down different forms of cholesterol, which build up in certain areas of the body and are characterized by fatty yellow nodules (tendon xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body.  Disease symptomatology is characterized by chronic diarrhea during infancy and cataracts in late childhood. When patients enter adulthood, they begin to demonstrate progressive neurological symptoms, which may include dementia, seizures, hallucinations, depression and difficulty with coordination and speech. At present there is no cure for CTX, only treatment to slow disease progression.

Anne Douar, Chief Operating Officer at Vivet Therapeutics, commented: “CTX is a progressive, neurodegenerative disorder, that if left untreated can lead to debilitating consequences and a poor prognosis effecting a patient’s lifespan. The funding granted as part of France Health Innovation Plan 2030 will enable Vivet to accelerate the development of a transformative and potentially curative treatment with our gene therapy product VTX-806 for patients with CTX.” The €4.9 million funding is from the “Innovations in biotherapies” framework of the France Health Innovation Plan 2030 and specifically the acceleration strategy Biotherapies – Bioproduction in innovative therapies.

Coordinated by the French Health Innovation agency, the funding, will be distributed over a three-year period and operated by Bpifrance. Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure CTX in patients. By reinstating the CYP27A1 gene activity, which is impaired in CTX patients, using an adeno-associated viruses (AAV) vector, Vivet aims to demonstrate a safe and feasible treatment option for CTX disease. The funding will support the CrisTauX program which will cover preclinical research and development activities of VTX-806 and includes the manufacturing process development within medicine good manufacturing practices rules and a clinical study aiming at identifying biomarkers for the effectiveness of treatment in patients under standard of care.

Vivet Therapeutics was founded in 2016 by CEO Dr Jean-Philippe Combal and CSO Dr Gloria Gonzalez-Aseguinolaza. The company is backed by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer, Columbus Venture Partners, Ysios Capital, Kurma Partners and Eurazeo.

02/02/2024

Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis

Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis

 Funding from French Government as part of the France Health Innovation Plan 2030

Vivet Therapeutics, a French clinical stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic disorders, announces the financing of €4.9 million received from the French government to advance the development of a gene therapy for the treatment of cerebrotendinous xanthomatosis (CTX). This rare autosomal recessive genetic disorder affects the body’s ability to metabolize cholesterol. Patients with CTX are unable to break down different forms of cholesterol, which build up in certain areas of the body and are characterized by fatty yellow nodules (tendon xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body.  Disease symptomatology is characterized by chronic diarrhea during infancy and cataracts in late childhood. When patients enter adulthood, they begin to demonstrate progressive neurological symptoms, which may include dementia, seizures, hallucinations, depression and difficulty with coordination and speech. At present there is no cure for CTX, only treatment to slow disease progression.

Anne Douar, Chief Operating Officer at Vivet Therapeutics, commented: “CTX is a progressive, neurodegenerative disorder, that if left untreated can lead to debilitating consequences and a poor prognosis effecting a patient’s lifespan. The funding granted as part of France Health Innovation Plan 2030 will enable Vivet to accelerate the development of a transformative and potentially curative treatment with our gene therapy product VTX-806 for patients with CTX.” The €4.9 million funding is from the “Innovations in biotherapies” framework of the France Health Innovation Plan 2030 and specifically the acceleration strategy Biotherapies – Bioproduction in innovative therapies.

Coordinated by the French Health Innovation agency, the funding, will be distributed over a three-year period and operated by Bpifrance. Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure CTX in patients. By reinstating the CYP27A1 gene activity, which is impaired in CTX patients, using an adeno-associated viruses (AAV) vector, Vivet aims to demonstrate a safe and feasible treatment option for CTX disease. The funding will support the CrisTauX program which will cover preclinical research and development activities of VTX-806 and includes the manufacturing process development within medicine good manufacturing practices rules and a clinical study aiming at identifying biomarkers for the effectiveness of treatment in patients under standard of care.

Vivet Therapeutics was founded in 2016 by CEO Dr Jean-Philippe Combal and CSO Dr Gloria Gonzalez-Aseguinolaza. The company is backed by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer, Columbus Venture Partners, Ysios Capital, Kurma Partners and Eurazeo.

02/02/2024

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Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis

NIH study shows how genes in retina get regulated during development

Genome topology map of human retina development lays foundation for understanding diverse clinical phenotypes in simple and complex eye diseases

Cell differentiation during retinal organoid development

This image metaphorically captures the cell differentiation process during retinal organoid development. Loose yarn representing uncompacted DNA is wound by a crochet hook around buttons representing nucleosomes and culminating in a tightly condensed ball of chromatin that forms an eye-like shape. ©Erina He, NIH Medical Arts

Researchers at the National Institutes of Health have mapped the 3D organization of genetic material of key developmental stages of human retinal formation, using intricate models of a retina grown in the lab. The findings lay a foundation for understanding clinical traits in many eye diseases, and reveal a highly dynamic process by which the architecture of chromatin, the DNA and proteins that form chromosomes, regulates gene expression. The findings were published in Cell Reports. “These results provide insights into the heritable genetic landscape of the developing human retina, especially for the most abundant cell types that are commonly associated with vision impairment in retinal diseases,” said the study’s lead investigator, Anand Swaroop, Ph.D., chief of the Neurobiology, Neurodegeneration, and Repair Laboratory at the National Eye Institute (NEI), part of NIH.

Using deep Hi-C sequencing, a tool used for studying 3D genome organization, the researchers created a high-resolution map of chromatin in a human retinal organoid at five key points in development. Genes are interspersed throughout long strands of DNA. Those DNA strands get packaged into chromatin fibers, which are spooled around histone proteins and then repeatedly looped to form highly compact structures that fit into the cell nucleus. All those loops create millions of contact points where genes encounter non-coding DNA sequences, such as super enhancers, promoters, and silencers that regulate gene expression. Long considered “junk DNA”, these non-coding sequences are now recognized to play a crucial role in controlling which genes get expressed in a cell and when. Studies of chromatin’s 3D architecture shed light on how these non-coding regulatory elements exert control even when their location on a DNA strand is remote from the genes they regulate.

At each of the five key retinal organoid developmental stages, billions of chromatin contact point pairs were sequenced and analyzed. The findings reveal a dynamic picture: Spatial organization of the genome within the nucleus is transformed during retinal development, facilitating expression of specific genes at key time periods. For example, at a stage when immature cells start developing retinal cell characteristics, chromatin contact points shift from a mostly proximal-enriched state to add more distal interactions. There also appears to be a hierarchy of compartments that organize the contact point interactions. Some of these compartments, called “A” and “B”, are stable, but others swap during development, which further serves to enhance or inhibit gene expression. “The datasets resulting from this research serve as a foundation for future investigations into how non-coding sections of the genome are relevant for understanding divergent phenotypes in single gene mutation (Mendelian) disorders, as well as complex retinal diseases,” Swaroop said.

The study was funded by the NEI Intramural Research Program (ZIAEY000450 and ZIAEY000546). NEI is part of the National Institutes of Health.

Reference: Qu Z, Batz Z, Singh N, Marchal C, Swaroop A, “Stage-specific dynamic reorganization of genome topology shapes transcriptional neighborhoods in developing human retinal organoids”.

12/12/2023

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