Gain Therapeutics Secures €2.5 Million Series A Financing to Advance SEE-Tx Platform for Rare Genetic Disorders

VCBeat New Medicine (WeChat Official Account: biobeat1) has learned from foreign media that biotechnology startup Gain Therapeutics recently completed a €2.5 million Series A financing round. The proceeds will be used to develop Site-Enhanced Enzyme Therapy (SEE-Tx), a treatment for rare genetic mutation disorders.

It is reported that this round of financing marks Gain Therapeutics’ first fundraising effort, comprising €1.1 million from VitaTech, €1 million from the Helsinn Investment Fund, and €400,000 from TiVenture.

Gain Therapeutics, founded in 2017, is a clinical-stage biotechnology company headquartered in Switzerland. The company is developing its advanced therapy platform, SEE-Tx, which enables the identification of brain-penetrant non-competitive pharmacological chaperones for the treatment of chronic neurodegenerative diseases.

SEE-Tx is a platform capable of identifying pharmacological chaperones that are non-competitive with natural substrates and can enhance protein activity. Mutations in wild-type gene sequences lead to protein misfolding, characterized by reduced stability and functional impairment of the affected proteins, thereby causing many rare diseases. Pharmacological chaperone molecules can target the mutation sites responsible for protein misfolding, restore the function of the misfolded proteins, and prevent disease progression.

Gain Therapeutics has leveraged its proprietary SEE-Tx technology platform to discover a series of non-competitive pharmacological chaperone molecules. These molecules can identify misfolded proteins and restore their enzymatic activity. This mechanism has been validated through the GLB1 gene, which is involved in the pathogenesis of GM1 gangliosidosis (a lysosomal storage disease) and Morquio B disease.

GM1 gangliosidosis is a hereditary lysosomal storage disorder that progressively destroys neurons in the brain and spinal cord. The disease is caused by mutations in the GLB1 gene and is inherited in an autosomal recessive pattern. Symptoms may include hepatosplenomegaly, coarsening of facial features, skeletal abnormalities, joint stiffness, abdominal distension, and muscle weakness. Currently, there is no pharmacological treatment for GM1 gangliosidosis; however, the SEE-Tx platform has identified pharmacological chaperones for this condition.

In addition, hereditary lysosomal storage disorders may also be caused by mutations in the GBA gene. This type of lysosomal storage disorder leads to the accumulation of harmful lipids in the patient’s body, resulting in Gaucher disease, accompanied by splenic and hepatic dysfunction.

Professor Xavier Barril, Chief Scientific Officer at Gain Therapeutics, stated, “This round of financing will support the advancement of our latest SEE-Tx programs, including research into gangliosidoses, Gaucher disease, and Parkinson’s disease associated with GLB1 and GBA gene mutations. Gain Therapeutics’ current objective is to accelerate the identification of clinical candidates to enable early testing in human studies.”

VitaTech primarily invests in early-stage and growth-stage high-tech companies in Europe, most of which belong to the healthcare industry. VitaTech is funded by the Zambon family, European high-net-worth private investors, and other family-owned businesses.

Backed by the Helsinn Group, the Helsinn Investment Fund focuses on early-stage investments in the healthcare sector, aiming to provide innovative technology companies with solutions to transform ideas into commercial ventures. The companies invested in by the Helsinn Investment Fund possess technologies across various fields, including metabolic regulation, cancer treatment, gastrointestinal diseases, and dermatological conditions.

TiVenture is the seed investment fund of Fondazione Centenario Banca Stato (Switzerland), which invests in early-stage companies with innovative technologies and high growth potential, primarily in Southern Switzerland. The firm maintains a diversified portfolio, with a primary focus on the life sciences, healthcare, high-tech, and IT sectors.

(Editor: Wang Chan)