RARE Daily

Gain Therapeutics Raises $40M in IPO to Advance Rare Disease Pipeline

March 18, 2021

Rare Daily Staff

Preclinical rare disease focused biotech Gain Therapeutics raised $40 million in an initial public offering of 3.6 million shares of its common stock at a price of $11.00 per share.

The shares will trade on the Nasdaq Global Market under the symbol “GANX.” The company also granted underwriters a 30-day option to purchase up to an additional half million shares of common stock at the initial public offering price, less underwriting discounts and commissions.

Gain Therapeutics uses its See-Tx target identification platform to identify and optimizing allosteric binding sites that have never before been targeted, in order to unlock new treatment options for difficult-to-treat disorders characterized by protein misfolding.

The biotech’s pipeline of small molecule therapies target lysosomal storage disorders and some neurodegenerative diseases such as Parkinson’s disease. Lead compounds target GM1 Gangliosidosis, an inherited progressive disease that impacts the neurons in the brain and spinal cord, and Gaucher disease, the most common lysosomal storage disease that is caused by mutations in GBA, which leads to the toxic buildup of fat in a variety of organs and tissues such as the liver, spleen, bones and central nervous system. While there is currently no cure for these patients, enzyme replacement therapy can be used to address non-CNS symptoms. Gain is developing allosteric regulators to potentially provide the first treatment approach for the disease’s neuronopathic symptoms.

The company has identified and filed three patent applications and one provisional patent application for its novel Structurally Targeted Allosteric Regulators (STARs). Gain expects to obtain additional preclinical data and commence IND-enabling studies for lead compound candidates in 2021.

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