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This NASDAQ is Unlocking New Treatments for Neurodegenerative and Lysosomal Storage Diseases: Gain Therapeutics, Inc. (NASDAQ: GANX)

This NASDAQ is Unlocking New Treatments for Neurodegenerative and Lysosomal Storage Diseases: Gain Therapeutics, Inc. (NASDAQ: GANX)

– Two Patents on Modulator Compounds for Treatment of CNS Disorders.
– Initial Public Offering Raised $46 Million in Gross Proceeds.
– Agreement with Zentalis Pharmaceuticals for New Cancer Treatments.
– Four New Independent BOD Members to Strengthen Leadership Team.
– Expects to Complete Proof of Concept Studies and Begin IND-enabling Trials in the Second Half of 2021.

Gain Therapeutics, Inc. (NASDAQ: GANX) is redefining drug discovery with its SEE-Tx(TM) target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, GANX is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding.

GANX was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse.

– Issuance of Two New PCT Patents Covering GBA and GLAC Modulator Compounds for Treatment of CNS Disorders

Activity of GBA and GALC Enzymes Play Key Roles in the Neurodegenerative Diseases and Lysosomal Storage Diseases

On June 10th GANX announced the publication of two PCT patents. PCT/IB2020/061158 and PCT/IB2020/0611156 are directed at compounds targeting misfolded beta-​glucocerebrosidase (GBA) and galactosylceramidase (GALC) respectively, addressing central nervous system (CNS) and demyelinating disorders such as Parkinson’s Disease, Lewy body dementia and Alzheimer’s.

The GANX patent PCT/IB2020/061158 covers compounds and therapeutic uses of conditions associated with the alteration of Beta-glucocerebrosidase (GBA). These conditions include Gaucher Disease, Parkinson’s Disease, and other Lewy body disorders including Alzheimer’s Disease and amyotrophic lateral sclerosis (ALS).

The GANX patent PCT/IB2020/0611156 covers compounds and therapeutic uses of conditions associated with the alteration of galactosylceramidase (GALC). These conditions include lysosomal storage diseases and other demyelinating diseases, such as Krabbe Disease, Lewy body dementia, Multiple Sclerosis, and peripheral neuropathy.

– GANX First Quarter 2021 Review

On May 10th GANX provided a corporate update for first quarter of 2021 which included the following highlights:

GANX multi-target collaboration agreement with Zentalis Pharmaceuticals –

In April 2021 GANX announced a multi-target collaboration agreement with Zentalis Pharmaceuticals (NASDAQ: ZNTL) to discover new product candidates for the treatment of cancer. GANX will use its proprietary SEE-Tx computational platform technology to identify new sites on target proteins for potential use in oncology.

GANX presentation of data at the International Association of Parkinsonism and Related Disorders 2021 World Congress –

In April 2021 GANX announced the presentation of a poster at the International Association of Parkinsonism and Related Disorders (IAPRD) 2021 World Congress being held virtually May 1-4, 2021. The poster highlights data demonstrating the potential of the GANX structurally targeted allosteric regulators (STARs) to restore relevant biological function in vitro and improve motor deficits in an in vivo model of Parkinson’s disease.

GANX Completed Successful IPO –

In March 2021 GANX completed an oversubscribed initial public offering (IPO) of 4,181,818 shares of common stock at a public offering price of $11.00 per share, including the full exercise by the underwriters of their overallotment option to purchase 545,454 additional shares of common stock. GANX received gross proceeds of approximately $46 million in the offering. This financing strengthened the GANX balance sheet it with significant funding to advance its therapeutics programs and pipeline expansion.

GANX presented pre-clinical data at the 17 th Annual WorldSymposium –

In February 2021 GANX presented positive pre-clinical data supportive of in vitro engagement for Gaucher disease, GM1 gangliosidosis, and Morquio B programs at the 17 th Annual WorldSymposium(TM).

GANX appointed new members to the board of directors with deep financial, clinical development and regulatory experience –

In February 2021 GANX strengthened its leadership team with the appointment of four bio-industry veterans to its Board of Directors; Dov Goldstein, M.D., Gwen Melincoff, Claude Nicaise, M.D., and Hans Peter Hasler.

For more information on Gain Therapeutics, Inc. (NASDAQ: GANX) visit

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