Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”),
a clinical-stage biotechnology company leading the discovery and
development of the next generation of allosteric small molecule
therapies, today announces the presentation of a poster at the
International Conference on Alzheimer's and Parkinson's Diseases
and Related Neurological Disorders (AD/PD™ 2024) that addresses the
mechanism of action of the Company’s lead compound, GT-02287.
AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon,
Portugal.
The poster, titled “GT-02287, a Clinical Stage
Glucocerebrosidase Regulator for the Treatment of PD, Eases ER
Stress and Enhances Lysosomal Enzyme Activity,” which will be
available online March 5th and presented on-site on the 8th and 9th
by Dr. Natalia Perez-Carmona, demonstrates how GT-02287, through
its interaction with glucocerebrosidase (GCase) in the endoplasmic
reticulum (ER), aids correct GCase folding, preventing ER
retention, ER stress, and ER-associated degradation of mutated
GCase enzyme. GCase is consequently able to travel to the lysosome,
resulting in enhanced lysosomal activity and efficient processing
of the GCase substrate glucosylceramide. Increase in GCase
substrate in the lysosome was previously shown to be associated
with accumulation of aggregated alpha-synuclein, a pathological
hallmark of Parkinson’s disease and related disorders. The study
was done in collaboration with scientists in the group of Professor
Maurizio Molinari at the Università dela Svizzera
italiana-affiliated Institute for Research in Biomedicine in
Bellinzona, Switzerland and was conducted under the support of a
Eurostars-2 joint program.
“These data further confirm our understanding of
the mechanism of action of GT-02287, how it impacts cellular health
by preventing the downstream consequences of GCase misfolding,
including cellular stress and lysosomal dysfunction” commented Dr.
Natalia Perez-Carmona, Senior Director of Biology at Gain
Therapeutics and presenting author. “The AD/PD™ conference is an
important opportunity for us to continue to discuss our
differentiated approach to addressing GBA1 Parkinson’s disease
through GCase modulation among this key audience.”
A PDF of the poster presented at the AD/PDTM 2024 conference is
available on the Science & Technology section of the Company’s
website
at https://www.gaintherapeutics.com/science-technology/posters.html.
The AD/PD™ Conference is an annual event
attracting leading medical and scientific professionals from around
the world, focusing on the advances of science and
therapy of Alzheimer’s and Parkinson’s Diseases and related
neurological disorders.
About GT-02287
Gain Therapeutics’ lead drug candidate,
GT-02287, is in clinical development for the treatment of GBA1
Parkinson’s disease (GBA1-PD). The orally administered,
brain-penetrant small molecule is an allosteric protein modulator
that restores the function of the lysosomal protein enzyme
glucocerebrosidase (GCase) which becomes misfolded and impaired due
to a GBA1 gene mutation, the most common genetic abnormality
associated with PD. In preclinical models of PD, GT-02287 restored
GCase enzymatic function, reduced aggregated α-synuclein,
neuroinflammation and neuronal death, increased dopamine levels and
improved motor function. Additionally, GT-02287 significantly
reduced plasma neurofilament light chain (NfL) levels, an emerging
biomarker for neurodegeneration.
Gain’s lead program in Parkinson’s disease 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 – Swiss Innovation Agency.
About GBA1 Parkinson’s Disease
GBA1 Parkinson’s disease is caused by mutations
in the GBA1 gene, found in up to 15% of patients with Parkinson’s
disease and making it the primary genetic risk factor. The mutation
causes dysfunctional misfolding of the lysosomal enzyme
glucocerebrosidase (GCase), reducing its activity in the brain and
leading to the subsequent accumulation of α-synuclein and
degeneration of dopamine-containing nerve cells. Patients with
GBA1-PD tend to have earlier onset and faster symptom progression
than those with sporadic PD, a progressive neurodegenerative
disease characterized by a motor syndrome consisting of
bradykinesia (slowness of movement), rigidity, resting tremors, and
postural instability. With current therapies treating only the
symptoms of Parkinson’s disease without affecting the underlying
progression of the disease, there is an unmet need to develop novel
disease-modifying therapies such as GT-02287 that have the
potential to slow or stop disease progression and help improve
outcomes in this patient population.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is a clinical-stage
biotechnology company leading the discovery and development of next
generation allosteric therapies. Gain’s lead drug candidate
GT-02287 for the treatment of GBA1 Parkinson’s disease, is
currently being evaluated in a Phase 1 clinical trial.
Leveraging AI-supported structural biology,
proprietary algorithms and supercomputer-powered physics-based
models, the company’s Magellan™ drug discovery platform can
identify novel allosteric binding sites on disease-implicated
proteins, pinpointing pockets that cannot be found or drugged with
current technologies. Magellan™ is the next generation of Gain’s
original SEE-Tx® (Site-Directed Enzyme Enhancement Therapy)
platform, which was enhanced and expanded with new AI and
machine-learning tools and virtual screening capabilities to access
the emerging on-demand compound libraries covering vast chemical
spaces of over 50 billion compounds.
Gain’s unique approach enables the discovery of
novel, allosteric small molecule modulators that can restore or
disrupt protein function. Deploying its highly advanced platform,
Gain is accelerating drug discovery and unlocking novel
disease-modifying treatments for untreatable or difficult-to-treat
disorders including neurodegenerative diseases, rare genetic
disorders and oncology. For more information, please visit
GainTherapeutics.com and follow us on LinkedIn.
About Institute For Research in
Biomedicine (IRB, Bellinzona)
Founded in 2000 in Bellinzona, Switzerland, the
Institute for Research in Biomedicine (IRB) hosts 13 research
groups studying infectious, inflammatory, rare diseases and tumors
in order to identify new therapeutic strategies. As a result of its
activities, the IRB has gained considerable international
recognition in the fields of immunology, cell and structural
biology and DNA repair. Molinari’s laboratory investigates the
molecular mechanisms regulating chaperone-assisted protein folding
and the quality control processes determining whether a polypeptide
should be transported at the intra- or extra-cellular site of
activity, or if it should be selected for degradation. Emphasis is
given on establishing causes of diseases resulting from misfolding
of mutant gene products such as α1-antitrypsin deficiency and
lysosomal storage disorders and to explore novel therapeutic
approaches to alleviate disease phenotypes. IRB offers training
opportunities for young researchers at all levels. This is made
possible through collaborations with Swiss and foreign
universities. Since its opening, IRB has trained more than 120 PhD
students who have gone on to careers in academia or industry both
in Switzerland and abroad. In 2021, IRB and IOR (Institute of
Oncology Research) formed the Bios+ (Bellinzona Institutes of
Science) association, with the mission to create new synergies and
to promote and coordinate the scientific research and teaching
activities of the two institutes. The vision of Bios+ is to promote
the growth of a biomedical research center in Italian-speaking
Switzerland. For more information, please visit www.irb.usi.ch.
Cautionary Note Regarding Forward-Looking
Statements
This press release contains "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995. All statements in this press release other than
statements of historical facts are “forward-looking statements”. In
some cases, you can identify these statements by forward-looking
words such as "may," "might," "will," "should," "expect," "plan,"
"anticipate," "believe," "estimate," "predict," "goal, " "intend,"
"seek, " "potential" or "continue," the negative of these terms and
variations of these words or similar expressions that are intended
to identify forward-looking statements, although not all
forward-looking statements contain these words. Forward-looking
statements in this press release include, but are not limited to,
statements regarding: the development of the Company’s current or
future product candidates including GT-02287; expectations
regarding the timing of results from a Phase 1 clinical study for
GT-02287; and the potential therapeutic and clinical benefits of
the Company’s product candidates. These forward-looking statements
are based on the Company’s expectations and assumptions as of the
date of this press release. Each of these forward-looking
statements involves risks and uncertainties that could cause the
Company’s preclinical and future clinical development programs,
future results or performance to differ materially from those
expressed or implied by the forward-looking statements. These
statements are not historical facts but instead represent the
Company's belief regarding future results, many of which, by their
nature, are inherently uncertain and outside the Company's control.
Many factors may cause differences between current expectations and
actual results, including the impacts of the post-COVID-19
environment and other global and macroeconomic conditions on the
Company’s business; clinical trials and financial position;
unexpected safety or efficacy data observed during preclinical
studies or clinical trials, clinical trial site activation or
enrollment rates that are lower than expected; changes in expected
or existing competition; changes in the regulatory environment; the
uncertainties and timing of the regulatory approval process; and
unexpected litigation or other disputes. Other factors that may
cause the Company’s actual results to differ from those expressed
or implied in the forward-looking statements in this press release
are identified in the section titled “Risk Factors,” in the
Company’s Annual Report on Form 10-K filed with the Securities and
Exchange Commission on March 23, 2023 and its other documents
subsequently filed with or furnished to the Securities and Exchange
Commission from time to time. All forward-looking statements
contained in this press release speak only as of the date on which
they were made. The Company undertakes no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date on which they were made, except as required by
law.
Investor Contact:
CORE IR(516) 222-2560 ir@gaintherapeutics.com
Media Contacts:
Russo PartnersNic Johnson and Elio
Ambrosionic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(212)
845-4242
Gain Therapeutics (NASDAQ:GANX)
과거 데이터 주식 차트
부터 4월(4) 2024 으로 5월(5) 2024
Gain Therapeutics (NASDAQ:GANX)
과거 데이터 주식 차트
부터 5월(5) 2023 으로 5월(5) 2024