The public-private consortium formed by the biotech company Connecta Therapeutics, the Centre for Genomic Regulation (CRG) and the Hospital del Mar Medical Research Institute (IMIM) has received a grant for 1,970,520 Euro from the Spanish National Innovation Agency to develop an innovative treatment for fragile X syndrome (FXS), the most common form of hereditary intellectual disability.

The grant comes from the 2019 “Collaboration Challenges” call for projects of the National R&D&I Programme geared towards societal challenges. The call received 420 applications, 158 of which will be funded.

In the first phase, the Cellular Neurobiology and Integrated Systems group in the Systems Biology programme of the CRG led by Dr Mara Dierssen will conduct complementary pharmacology studies on Connecta Therapeutics CTH120 molecule. Dr Dierssen highlights the synergies between Connecta, the CRG and the IMIM: “This project brings together a multidisciplinary team of scientists and entrepreneurs with in-depth knowledge and experience in the neuropharmacology of intellectual disability with the goal of attaining a real scientific breakthrough to respond to the challenges society faces”.

Later, the IMIM coordinated by Dr Rafael de la Torre, director of the Neuroscience Research Programme and leader of the Integrative Pharmacology and Systems Neuroscience Research group, will start the Phase I clinical trial (expected for early 2022). “FXS is the leading cause of hereditary intellectual disability. Although there are different degrees of severity, the impact of the diagnosis causes a profound crisis in families” he comments.

Connecta Therapeutics will lead the project. The company specializes in treatments for unmet medical needs concerning the central nervous system. Dr Josep Prous, the biotech’s co-founder and CSO, highlights that “CTH120 is a first-in-class drug that will be able to address the disease based on an innovative strategy as it is capable of modulating neuroplasticity, targeting the pathophysiological origin of fragile X syndrome”.

At the end of September 2020, Connecta Therapeutics closed a 1.7 million Euro round of investment led by the venture capital company Inveready which also featured the CDTI through its Innvierte programme as well as the company’s co-founders.

Fragile X Syndrome

FXS is a hereditary genetic disorder linked to the X chromosome that causes intellectual disability and emotional and social problems ranging from mild to severe issues, such as hyperactivity, anxiety, aggressive behaviours, and autism. Its prevalence is estimated between 1/3,600-4,000 in men and 1/4,000-6,000 in women. The syndrome is diagnosed in newborns or in childhood through DNA blood tests, but there is no specific treatment directed at the cause of the disease, only treatments that help relieve its symptoms.

The drug CTH120

FXS is due to a mutation that leads to a FMRP protein deficiency, which has an important role in the plasticity and maturation of synaptic connections between neurons, facts that seem to be related with the intellectual disability and symptoms of those affected. Preclinical studies have demonstrated that the drug CTH120 by Connecta Therapeutics is a good neuroplasticity modulator. At the same the drug has been shown to enhance cognitive capacity and improve autistic signs and symptoms such as social interaction issues.

About Connecta Therapeutics

Connecta Therapeutics is a biotechnology company that is developing new treatments for unmet medical needs involving the central nervous system. It is initially focused on developing a first-in-class drug to treat fragile X syndrome. Founded in Barcelona in 2019 by Prous Institute for Biomedical Research, Josep Prous, David Prous and Jordi Fàbrega. Its headquarters are located at the Barcelona Science Park.

About the CRG

The Centre for Genomic Regulation (CRG) is a renowned international biomedical research centre located in Barcelona. Its mission is to discover and promote knowledge to benefit society, public health, and economic prosperity. The centre employs more than 400 interdisciplinary scientists focused on understanding the complexity of life, from the genome to the cell and the entire organism. The CRG forms part of The Barcelona Institute of Science and Technology (BIST) and is a CERCA research centre of the Government of Catalonia.

About the IMIM

The Hospital del Mar Medical Research Institute (IMIM) of Barcelona is a research centre in biomedicine and health sciences, organised across five large programmes: cancer, epidemiology and public health, biomedical computer sciences, neurosciences, and inflammatory and cardiovascular processes. Featuring around 700 professionals, it is among the top ten Spanish institutions in terms of scientific impact on healthcare. It is a CERCA research centre of the Government of Catalonia and is accredited by the Carlos III Health Institute.

Fuente: Connecta Therapeutics
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