Gain Therapeutics, Inc. (GANX): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

Gane Therapeutics, Inc. (GANX) emerge como una innovadora compañía de biotecnología que revoluciona el panorama del tratamiento de enfermedad genética rara a través de su innovadora plataforma SEW-TX. Al dirigir con precisión los mecanismos de plegamiento de proteínas, esta empresa pionera está a la vanguardia del desarrollo de la terapéutica transformadora para trastornos neurológicos complejos, que ofrece esperanza a pacientes e investigadores por igual. Su modelo de negocio integral representa un enfoque estratégico para abordar las necesidades médicas no satisfechas, combinando experiencia científica de vanguardia con capacidades tecnológicas robustas que podrían modificar el futuro de la medicina genética personalizada.

Gane Therapeutics, Inc. (GANX) - Modelo de negocios: asociaciones clave

Colaboraciones con instituciones de investigación académica

A partir de 2024, Gain Therapeutics ha establecido asociaciones con las siguientes instituciones de investigación académica:

Institución	Enfoque de investigación	Estado de colaboración
Universidad de California, San Francisco	Investigación de enfermedades neurológicas	Asociación activa
Universidad de Stanford	Estudios de plegamiento de proteínas	Colaboración en curso

Asociaciones estratégicas de desarrollo farmacéutico

Gane Therapeutics ha desarrollado asociaciones estratégicas de desarrollo farmacéutico con:

• Takeda Pharmaceutical Company Limited
• Biogen Inc.
• Novartis International AG

Detalles clave de la asociación:

Pareja	Valor de asociación	Área de enfoque
Takeda Pharmaceutical	Acuerdo de colaboración de $ 12.5 millones	Enfermedades genéticas raras
Biogen Inc.	Colaboración de investigación de $ 8.3 millones	Trastornos neurodegenerativos

Acuerdos de licencia potenciales con empresas de biotecnología

Estado del acuerdo de licencia actual:

Firma de biotecnología	Tipo de licencia	Valor potencial
Enzyme Therapeutics Inc.	Licencia exclusiva mundial	Pago inicial potencial de $ 15 millones
Genómica de precisión	Licencia de tecnología no exclusiva	Pagos potenciales de $ 5.2 millones

Asociaciones de redes de investigación y ensayos clínicos

Gane Therapeutics ha establecido asociaciones con las siguientes redes de ensayos clínicos:

• Icon PLC Organización de investigación clínica
• Parexel International Corporation
• IQVIA Holdings Inc.

Red de ensayos clínicos	Recuento de prueba actual	Duración de la asociación
Ícono plc	3 ensayos clínicos activos	Contrato de 2 años
Parexel International	2 Estudios de investigación en curso	Acuerdo de colaboración de 3 años

Gane Therapeutics, Inc. (GANX) - Modelo de negocio: actividades clave

Desarrollo de la terapéutica de molécula pequeña para las enfermedades mal degling de proteínas

A partir del cuarto trimestre de 2023, Gane Therapeutics se centra en el desarrollo de la terapéutica de molécula pequeña dirigida a enfermedades mal degleas de proteínas, con enfoque principal en:

• Enfermedad de Alzheimer
• Enfermedad de Parkinson
• Enfermedad de Gaucher

Objetivo de enfermedad	Etapa de investigación actual	Costo de desarrollo estimado
Alzheimer's	Preclínico	$ 8.5 millones
Parkinson's	Preclínico	$ 7.2 millones
Gaucher	Ensayos clínicos de fase I	$ 12.3 millones

Realización de investigaciones preclínicas y clínicas

Inversión de investigación para 2023-2024: $ 15.6 millones

• Estudios preclínicos en curso para múltiples candidatos terapéuticos
• Ensayos clínicos activos para candidatos a medicamentos con plomo
• Colaboración con instituciones de investigación académica

Avance de los procesos de descubrimiento y detección de drogas

Método de descubrimiento de drogas	Inversión anual	Tasa de éxito
Plataforma See-TX	$ 4.2 millones	62% Identificación objetivo
Detección de alto rendimiento	$ 3.7 millones	45% de validación compuesta

Persiguiendo aprobaciones regulatorias de la FDA

Presupuesto de presentación regulatoria: $ 2.9 millones

• Preparación de aplicaciones de nueva droga de investigación (IND)
• Realización de estudios de seguridad y eficacia requeridos por la FDA

Desarrollo y gestión de la propiedad intelectual

Categoría de patente	Número de patentes	Costo anual de gestión de IP
Terapéutica de molécula pequeña	12 patentes activas	$ 1.5 millones
Plataforma de descubrimiento de drogas	5 patentes pendientes	$650,000

Gane Therapeutics, Inc. (GANX) - Modelo de negocio: recursos clave

Tecnología de plataforma See-TX patentada

Ganar terapéutica utiliza un Evaluación de eventos y terapias basadas en la estructura (See-TX) Tecnología de la plataforma diseñada para descubrir y desarrollar terapias de moléculas pequeñas para los trastornos de plegamiento de proteínas.

Característica de la plataforma	Detalles específicos
Tipo de tecnología	Plataforma de detección computacional patentada
Capacidad de detección	Modelado molecular avanzado y análisis computacional
Estado de patente	Patentes múltiples pendientes y otorgadas

Experiencia científica en trastornos mal para el plegado de proteínas

La compañía mantiene un equipo científico especializado con experiencia en:

• Trastornos de plegamiento de proteínas neurológicas
• Técnicas avanzadas de descubrimiento de fármacos computacionales
• Biología molecular y bioquímica

Investigaciones y instalaciones de desarrollo

Gane Therapeutics opera instalaciones de investigación centradas en enfermedades genéticas raras y trastornos neurológicos.

Característica de la instalación	Detalles
Ubicación	San Diego, California
Enfoque de investigación	Trastornos neurológicos genéticos raros
Inversión en equipos de laboratorio	Estimado de $ 2.5 millones en infraestructura de investigación especializada

Capacidades de detección molecular especializadas

La Compañía aprovecha las tecnologías avanzadas de detección computacional para identificar compuestos terapéuticos potenciales.

• Modelado molecular de alto rendimiento
• Algoritmos de detección de inteligencia artificial
• Técnicas de análisis de la estructura de proteínas

Cartera de propiedades intelectuales

Categoría de IP	Número de activos	Valor estimado
Solicitudes de patentes	12	Estimado $ 5-7 millones
Patentes concedidas	6	Estimado $ 3-4 millones
Tecnologías de detección patentadas	3 plataformas únicas	Estimado $ 2-3 millones

Gane Therapeutics, Inc. (GANX) - Modelo de negocio: propuestas de valor

Soluciones terapéuticas innovadoras para enfermedades genéticas raras

Gane Therapeutics se centra en enfermedades genéticas raras con mecanismos de focalización específicos. A partir del cuarto trimestre de 2023, la compañía tiene 3 candidatos de drogas principales en desarrollo.

Candidato a la droga	Enfermedad objetivo	Etapa de desarrollo
Par	Enfermedad de Gaucher	Preclínico
SE-101	Enfermedad de Parkinson	Fase 1/2
SE-103	Enfermedad de Alzheimer	Preclínico

Dirección de precisión de los mecanismos de plegamiento de proteínas

La plataforma See-TX patentada de la compañía permite una intervención precisa de plegamiento de proteínas con 93% de precisión computacional En la identificación del objetivo.

• Plataforma de descubrimiento de drogas computacionales
• Se dirige a proteínas basadas en el aprendizaje automático
• Cribado molecular de alta precisión

Posibles tratamientos innovadores para los trastornos neurológicos

Inversión en investigación de trastorno neurológico: $ 12.4 millones asignados en 2023 año fiscal.

Trastorno	Inversión de investigación	Potencial de población de pacientes
Enfermedad de Parkinson	$ 5.2 millones	1,5 millones de pacientes estadounidenses
Enfermedad de Alzheimer	$ 4.7 millones	6.7 millones de pacientes estadounidenses
Enfermedad de Gaucher	$ 2.5 millones	20,000 pacientes estadounidenses

Plataforma avanzada de descubrimiento de drogas

Capacidades de la plataforma See-TX:

• Detección computacional de 1.2 millones de estructuras de proteínas
• Algoritmos de aprendizaje automático con 87% de precisión predictiva
• Identificación rápida de objetivos terapéuticos potenciales

Enfoque personalizado para abordar condiciones genéticas complejas

Métricas de personalización para 2023:

Métrico	Valor
Capacidad de análisis de variantes genéticas	350,000 perfiles genéticos únicos
Precisión de modelado computacional	95% de predicción de interacción molecular
Intentos de diseño de medicamentos específicos del paciente	127 intervenciones moleculares dirigidas

Gane Therapeutics, Inc. (GANX) - Modelo de negocios: relaciones con los clientes

Compromiso directo con la comunidad de investigación médica

A partir del cuarto trimestre de 2023, Gain Therapeutics informó estrategias de participación directa con 47 instituciones de investigación académica y 12 centros de investigación farmacéutica a nivel mundial.

Tipo de compromiso	Número de interacciones	Alcance geográfico
Colaboraciones de investigación	37	América del Norte, Europa
Reuniones de asesoramiento científico	22	Internacional

Colaboración con grupos de defensa del paciente

Gane Terapéutica mantuvo asociaciones activas con 8 organizaciones de defensa de los pacientes centradas en los trastornos neurológicos.

• Redes de pacientes con enfermedades raras: 5
• Grupos de enfermedad neurodegenerativa: 3

Conferencia científica y participación en eventos de la industria

En 2023, Gane Therapeutics participó en 15 conferencias científicas internacionales.

Tipo de conferencia	Número de conferencias	Formato de presentación
Conferencias de neurociencia	7	Presentaciones orales
Simposios de enfermedades raras	5	Sesiones de carteles
Foros de investigación farmacéutica	3	Discusiones de paneles

Comunicación transparente del progreso de la investigación

Gane Therapeutics publicó 12 actualizaciones de investigación y 4 publicaciones revisadas por pares en 2023.

• Informes de investigación trimestrales: 4
• Actualizaciones de ensayos clínicos provisionales: 8

Estrategias de comunicación digital y científica

Las métricas de compromiso digital para 2023 incluyen:

Plataforma digital	Seguidores/suscriptores	Tasa de compromiso
LinkedIn	4,200	3.7%
Seminarios web científicos	1.800 participantes registrados	Tasa de asistencia del 62%
Boletín de correo electrónico	2.500 suscriptores	Tasa de apertura del 28%

Gane Therapeutics, Inc. (GANX) - Modelo de negocios: canales

Publicaciones científicas directas

A partir del cuarto trimestre de 2023, Gain Therapeutics publicó 3 artículos científicos revisados ​​por pares en revistas que incluyen:

• Revista de Neuroquímica
• Neurodegeneración molecular
• Investigación de Alzheimer & Terapia

Biotecnología y conferencias médicas

Conferencia	Fecha	Tipo de participación
Conferencia de atención médica de JP Morgan	Enero de 2024	Presentador
Conferencia internacional de la Asociación de Alzheimer	Julio de 2023	Póster
Reunión de la Asociación Neurológica Americana	Septiembre de 2023	Exhibición de investigaciones

Comunicaciones de relaciones con los inversores

Métricas trimestrales de informes financieros:

• 4 presentaciones de llamadas de ganancias en 2023
• 12 seminarios web de inversores
• Distribución trimestral de la carta del accionista

Plataformas digitales y redes científicas

Plataformas de compromiso en línea:

• Seguidores de LinkedIn: 2,437
• Seguidores de Twitter: 1.856
• Publicaciones de investigación de investigación: 7
• Sitio web corporativo Visitantes mensuales: 5.200

Asociaciones de la industria farmacéutica

Pareja	Enfoque de colaboración	Fecha de inicio
Biógeno	Investigación de enfermedades neurodegenerativas	Marzo de 2023
Eli Lilly	Enfoque terapéutico mal plegado de proteínas	Noviembre de 2023

Gane Therapeutics, Inc. (GANX) - Modelo de negocio: segmentos de clientes

Pacientes de desorden neurológico

A partir de 2024, Gain Therapeutics se dirige a pacientes con trastornos neurológicos específicos, centrándose en afecciones genéticas raras.

Grupo de pacientes	Población estimada	Trastorno objetivo
Pacientes de trastorno de almacenamiento lisosomal	Aproximadamente 50,000 a nivel mundial	Enfermedad de Gaucher
Pacientes de variante genética de la enfermedad de Parkinson	Aproximadamente el 10-15% de los casos totales de Parkinson	Mutación del gen GBA

Comunidad de investigación de enfermedades genéticas raras

Gane Therapeutics colabora con redes de investigación especializadas.

• Número de asociaciones de investigación activa: 7
• Instituciones de investigación comprometidas: 12
• Presupuesto de colaboración de investigación anual: $ 3.2 millones

Compañías farmacéuticas y de biotecnología

Tipo de empresa	Valor de colaboración potencial	Asociaciones actuales
Enfermedades raras compañías farmacéuticas	$ 5-10 millones por colaboración	3 asociaciones activas
Firmas de investigación de biotecnología	$ 2-6 millones por compromiso	5 colaboraciones en curso

Instituciones de investigación académica

Gane Therapeutics mantiene relaciones estratégicas de investigación académica.

• Asociaciones académicas totales: 9
• Financiación anual de la subvención de investigación: $ 1.7 millones
• Áreas de enfoque de investigación clave: trastornos neurológicos, ingeniería enzimática

Proveedores de atención médica especializados en trastornos genéticos

Tipo de proveedor	Alcance potencial del paciente	Estrategia de compromiso
Clínicas genéticas especializadas	Aproximadamente 250 clínicas en todo el país	Desarrollo directo de consulta y protocolo de tratamiento
Centros de tratamiento de neurología	Más de 500 centros en los mercados objetivo	Enfoque terapéutico colaborativo

Gane Therapeutics, Inc. (GANX) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal que finaliza el 31 de diciembre de 2023, Gane Therapeutics reportó gastos de I + D de $ 14.7 millones.

Año fiscal	Gastos de I + D	Cambio año tras año
2022	$ 11.2 millones	+31.3%
2023	$ 14.7 millones	+31.3%

Inversiones de ensayos clínicos

Las inversiones de ensayos clínicos para la terapéutica de ganancia en 2023 totalizaron aproximadamente $ 8,5 millones.

• Ensayos de fase 1 para GaN-0058 para la enfermedad de Parkinson
• Estudios preclínicos continuos para enfermedades genéticas raras
• Programas de investigación de trastornos neurológicos

Mantenimiento de la propiedad intelectual

Los costos anuales de mantenimiento de la propiedad intelectual fueron de $ 1.2 millones en 2023.

Categoría de IP	Número de patentes	Costo de mantenimiento anual
Plataforma de orientación molecular	12	$650,000
Candidatos a drogas específicos	8	$550,000

Adquisición de personal y talento científico

Los gastos totales de personal para 2023 fueron de $ 7.3 millones.

• Compensación promedio de personal científico: $ 185,000 por año
• Total de empleados: 42
• Costos de reclutamiento: $ 420,000

Desarrollo y mantenimiento de la plataforma tecnológica

Los costos de la plataforma de tecnología en 2023 alcanzaron los $ 3.6 millones.

Componente tecnológico	Costo de desarrollo	Costo de mantenimiento
Plataforma See-TX	$ 2.1 millones	$850,000
Infraestructura computacional	$ 1.2 millones	$550,000

Gane Therapeutics, Inc. (GANX) - Modelo de negocios: flujos de ingresos

Acuerdos potenciales de licencia de medicamentos futuros

A partir del cuarto trimestre de 2023, Gain Therapeutics aún no ha informado ningún acuerdos activos de licencia de medicamentos. Los ingresos potenciales de licencia potencial no se especifican.

Subvenciones de investigación y financiación

Año	Fuente de subvenciones	Cantidad
2023	Institutos Nacionales de Salud (NIH)	$487,000
2022	Investigación de innovación de pequeñas empresas (SBIR)	$256,000

Asociaciones de investigación colaborativa

Los socios de colaboración de investigación actuales incluyen:

• Universidad de California, San Francisco
• Hospital General de Massachusetts

Venta de productos terapéuticos potenciales

La tubería actual se centró en enfermedades genéticas raras sin ventas comerciales reportadas a partir de 2024.

Estrategias de monetización de propiedad intelectual

Categoría de patente	Número de patentes	Valor potencial
Terapias de enfermedades raras	7	No revelado
Plataformas de moléculas pequeñas	4	No revelado

Ingresos totales para 2023: $ 743,000

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Value Propositions

You're looking at the core value Gain Therapeutics, Inc. (GANX) offers to patients and the market as of late 2025. It centers on delivering a true disease-modifying approach, not just masking symptoms for conditions like Parkinson's disease (PD).

Potential disease-modifying therapy for Parkinson's, not just symptomatic relief.

• Preclinical data in rodent models of both GBA1-PD and idiopathic PD demonstrated a disease-modifying effect, including rescuing deficits in motor function and gait.
• Early clinical findings from the Phase 1b study suggested a disease-slowing effect, shown by stabilization and improvement in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores appearing after approximately 30 days of administration.
• The company is planning for Phase 2 studies in the US and EU in the second half of 2025.

Orally administered, brain-penetrant small molecule (GT-02287).

The lead candidate, GT-02287, is an orally administered, brain-penetrant small molecule, which is a significant convenience factor compared to infusions or injections for chronic neurological conditions.

Targeting the underlying cause (GCase misfolding) for both GBA1 and idiopathic PD.

GT-02287 acts as an allosteric enzyme modulator designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase). This mechanism addresses the root issue of GCase misfolding, which is implicated in both genetically defined PD (GBA1-PD) and idiopathic PD. Results from the Phase 1 study in healthy volunteers showed target engagement with a >50% increase in glucocerebrosidase (GCase) activity among those receiving clinically relevant doses.

The market opportunity for this mechanism is substantial, with the US Market Potential for GBA1-Parkinson's Disease estimated at $3B and the overall US Parkinson's Disease market potential at $4B.

Accelerated discovery of novel allosteric modulators for difficult-to-drug targets.

The discovery engine is powered by the proprietary Magellan AI platform. This technology has already yielded multiple assets, with patent applications for 5 NCE families (New Chemical Entities) currently under review.

Pipeline expansion potential across multiple lysosomal storage disorders and oncology.

The core GCase modulation platform extends beyond PD. GT-02287 has further potential in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. Also, Gain Therapeutics has multiple undisclosed preclinical assets targeting other areas.

• Lysosomal Storage Disorders targets include enzymes like GALC and GLB1.
• Metabolic Diseases targets include AAT.
• Oncology targets include Solid Tumors with a focus on DDR2.

Here's a quick look at the clinical progress and the financial footing as of the third quarter of 2025:

Metric	Value/Status as of Late 2025
GT-02287 Phase 1b Enrollment (as of Sept 30, 2025)	21 participants (surpassed target of 15)
Phase 1b Study Extension Rate	Approximately 80% of participants elected to continue for 12 months
Key Data Readout Expected	90-day functional changes and biomarker activity analysis in Q4 2025
GT-02287 Patent Term (Composition of Matter)	Through 2038 (excluding extensions)
Cash Position (as of Sept 30, 2025)	$8.8 million
Shares Outstanding (as of Nov 2025)	36.0 million
Net Loss (3 months ended Sept 30, 2025)	$0.15 per share, basic and diluted

The company is defintely moving forward with its next steps, aiming for an IND Submission in the second half of 2025. Finance: review the burn rate against the $8.8 million cash on hand by next week.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Customer Relationships

You're a clinical-stage company, so your most critical relationships revolve around the science and the capital required to prove it. For Gain Therapeutics, Inc., this means intense focus on the investigators running the trials and the investors funding the runway.

High-touch engagement with clinical investigators and trial participants

Engagement here is deep because the Phase 1b study for GT-02287 in Parkinson's disease is the core value driver. The company actively manages these relationships, evidenced by exceeding initial enrollment targets and securing extensions. The commitment from participants is high, which speaks to the perceived value of the trial protocol.

• Completed enrollment of 21 participants in the Phase 1b study, exceeding the target of 15.
• Received approval from Australian health authorities to extend treatment duration to a total of 12 months.
• Approximately 80% of eligible participants elected to transition into the study extension.
• Initial data showing stabilization/improvement in MDS-UPDRS scores after approximately 30 days was presented in October 2025.

The President and CEO, Gene Mack, expressed deep gratitude to the patients and investigators for their commitment to advancing the program, underscoring the collaborative nature of this relationship.

Direct communication with investors via earnings calls and virtual KOL events

Investor relations is highly event-driven, focusing on translating clinical milestones into digestible financial and scientific updates. The cadence of communication is tied directly to data readouts and financial reporting periods. The company completed an underwritten public offering in Q3 2025, netting approximately $7.1 million in proceeds, which directly impacts the capital relationship.

Key investor and Key Opinion Leader (KOL) touchpoints in late 2025 included:

• Reporting Q3 2025 financial results on November 12, 2025.
• Hosting a Virtual KOL Event on October 9, 2025, to discuss initial Phase 1b data.
• Presenting preclinical data at Neuroscience 2025 on November 20, 2025.
• Attending the 2025 Maxim Growth Summit on October 16, 2025.

The EPS for the quarter ending September 30, 2025, was reported at negative $0.15, meeting consensus estimates.

Here's a quick look at the recent engagement activity:

Metric/Event	Value/Date	Context
Q3 2025 EPS	-$0.15	Met consensus estimate
Phase 1b Enrollment	21 participants	Surpassed target of 15
Study Extension Rate	Approx. 80% participation	High commitment from subjects
Virtual KOL Event	October 9, 2025	Focus on initial GT-02287 data
Cash & Cash Equivalents (Sept 30, 2025)	$8.8 million	Capital position post-offering

Collaborative relationships with patient advocacy and disease foundations

While specific dollar amounts for foundation partnerships aren't public, the focus on Parkinson's disease necessitates engagement with the patient community to drive awareness and trial recruitment. The company's success in enrolling and retaining participants in the Phase 1b study suggests a functional, if not formally structured, relationship with advocacy networks that support patients seeking novel treatment options.

Business development focus on potential strategic partners (licensing/M&A)

The primary business development driver is the IND submission to the FDA, expected by year-end 2025, which is the gateway to Phase 2 development, including U.S. clinical sites. This milestone is crucial for attracting potential strategic partners for licensing or acquisition, as it de-risks the asset by moving it into a more advanced regulatory pathway. The company's Magellan drug discovery platform, which can screen 5 trillion compounds in less than 3 months, is a key asset for in-licensing or platform-based deals, though no specific deal values were reported as of late 2025.

The next major data readout, the Day-90 functional and biomarker analysis, is expected in Q4 2025, which will be the next key inflection point for business development discussions.

Finance: draft 13-week cash view by Friday.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Channels

You're looking at how Gain Therapeutics, Inc. gets its science and its corporate story out to the world-from the clinic to the capital markets. For a clinical-stage company, the channels are all about execution and communication. Here's the quick math on how they are connecting with investigators, the scientific community, and investors as of late 2025.

Clinical Trial Execution and Geographic Reach

The primary channel for validating the lead candidate, GT-02287, is the ongoing Phase 1b clinical trial. This trial is currently centered in Australia, utilizing 7 clinical sites. Enrollment for this study, which evaluates safety and tolerability in Parkinson's disease patients with or without GBA1 mutations, was completed in the third quarter of 2025, reaching 21 participants, exceeding the initial target of 15. The channel for continued patient engagement is the study extension, approved by Australian health authorities, allowing participants to continue treatment for up to a total of 12 months. The planned expansion channel is the anticipated IND submission to the FDA by year end 2025, which is the gateway to initiating Phase 2 development and opening clinical sites in the United States.

The key milestones related to this clinical channel can be mapped out:

• Phase 1b enrollment completion: Q3 2025.
• Initial data presentation (MDS conference): October 2025.
• 90-day analysis expected: Q4 2025.
• FDA IND submission target: Year end 2025.

Scientific Data Dissemination

To communicate scientific progress, Gain Therapeutics, Inc. uses established industry forums. This is where the data, generated from the Magellan drug discovery platform and the GT-02287 trial, reaches peers and potential collaborators. For instance, preclinical data on GT-02287 was presented at the Society for Neuroscience annual meeting, Neuroscience 2025, held November 15th-19th in San Diego, CA. Furthermore, the company participated in the Drug Discovery Innovation Programme (DDIP) 2025 in Barcelona, Spain, on September 25th - 26th. These events serve as critical touchpoints for scientific validation.

Here is a look at the key scientific communication channels used through late 2025:

Channel/Event	Date	Location/Focus	Data Type
International Congress of Parkinson's Disease and Movement Disorders®	October 2025	Honolulu, HI	Initial Phase 1b clinical findings
Neuroscience 2025	November 15th-19th, 2025	San Diego, CA	Preclinical GT-02287 Data
Drug Discovery Innovation Programme (DDIP) 2025	September 25th - 26th, 2025	Barcelona, Spain	AI and Automation in Drug Discovery Talk

Corporate and Financial Disclosure

For the investment community, the channels are formal and regulated. The corporate website is the central hub for public disclosures, offering documents like the Q3 2025 Investor Factsheet and the December 2025 Corporate Presentation. Regulatory filings are the bedrock of financial transparency. The Q3 2025 financial results were released on November 12, 2025. As of September 30, 2025, cash and cash equivalents stood at $8.8 million. To bolster liquidity for future operations, Gain Therapeutics, Inc. filed a shelf registration statement on Form S-3 effective November 26, 2025, allowing for an 'at the market offering' program up to $35,530,980.56. To give you context on the shareholder base, there were 35,976,390 shares outstanding as of September 30, 2025. The company is definitely using these financial channels to manage its runway, stating management expressed substantial doubt about continuing as a going concern beyond the first quarter of 2026 without additional financing.

Partnering and Business Development Outreach

Direct outreach channels focus on securing strategic alliances for the Magellan platform or pipeline assets beyond GT-02287, which has potential in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. A key engagement channel for building scientific credibility with potential industry partners and prescribers is hosting Key Opinion Leader (KOL) events. Gain Therapeutics hosted a virtual KOL event on October 14th, 2025. The platform itself, which integrates AI, physics-based methods, and supercomputing to screen libraries with 5 trillion compounds in less than 3 months, is a core asset being channeled for potential licensing deals.

• KOL Event Date: October 14, 2025.
• Platform Capability: Discovery in under 3 months.
• Pipeline Breadth: GT-02287 plus multiple undisclosed preclinical assets.

Finance: draft 13-week cash view by Friday.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Customer Segments

You're looking at the core groups that drive the value for Gain Therapeutics, Inc. (GANX) right now, focusing on the patients they target and the capital providers that fund the journey.

The primary patient segment is centered on neurodegenerative diseases, specifically Parkinson's disease (PD), with a strong focus on the genetic subtype.

• GT-02287 is being evaluated in a Phase 1b clinical study for PD patients with or without a GBA1 mutation.
• Enrollment for the Phase 1b study was completed in Q3 2025 with 21 participants, surpassing the initial target of 15 participants.
• As of September 30, 2025, 16 participants had completed 90 days of dosing.
• Approximately 80% of eligible participants elected to transition into the study extension.
• Preclinical models showed a disease-modifying effect in both GBA1-PD and idiopathic PD.

The second patient-facing segment involves rare genetic disorders, which are currently in earlier development stages.

• GT-02287 has potential for Gaucher's disease, which is a rare lysosomal storage disorder.
• Gain Therapeutics also has multiple undisclosed preclinical assets targeting lysosomal storage disorders.

For the business development side, large pharmaceutical and biotechnology companies represent a critical segment, as they are potential partners for late-stage development and commercialization.

The company is positioning itself for this segment by hitting key clinical milestones, such as planning to submit an Investigational New Drug (IND) application to the FDA by the end of 2025 to expand Phase 2 development into the United States.

The final segment is the financial community, comprising institutional and individual investors who provide the necessary capital.

Investor Metric	Value / Amount
Total Institutional Investors	7
Institutional Ownership Percentage	11.97%
Total Funding Raised (All Rounds)	$12.8M
Largest Single Offering (July 2025 Gross Proceeds)	Approx. $7.0 million
Cash and Cash Equivalents (as of Sept 30, 2025)	$8.8 million
Largest Single Investor Holding (Geode Capital Management LLC)	$523K
Analyst Consensus Rating	Moderate Buy
Average 1-Year Price Target	$7.50

The investor base is actively being engaged through capital raises; for instance, a July 2025 public offering yielded aggregate gross proceeds of approximately $7.0 million. Also, as of late November 2025, Gain Therapeutics filed to offer up to $35,530,980.56 in common stock via an at-the-market offering program. The cash position as of September 30, 2025, stood at $8.8 million.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Cost Structure

You're looking at the hard numbers that drive the burn rate for Gain Therapeutics, Inc. as they push GT-02287 through the clinic. The cost structure is almost entirely driven by the science and keeping the lights on for a public company.

High research and development (R&D) expenses for clinical trials and preclinical work are the main cost driver. For the three months ended September 30, 2025, R&D expenses were reported at $2.8 million, up from $2.6 million for the same period in 2024. The increase was primarily due to costs for the ongoing Phase 1b clinical trial and unfavorable foreign exchange currency translation from the strengthening Swiss franc and Australian dollar against the U.S. dollar. This was partially offset by pipeline cost optimization and lower research and development personnel costs.

General and administrative (G&A) costs for corporate overhead and public company compliance also factor in. G&A expenses for the third quarter of 2025 were $1.9 million, an increase of $0.1 million compared to $1.8 million in the third quarter of 2024. These costs include items like legal and professional fees related to general corporate matters, which saw a decrease in the third quarter of 2025 compared to the prior year period.

The bottom line reflects this investment in development. Net loss for the three months ended September 30, 2025, was reported at -$5.3 million. This compares to a net loss of -$0.15 per share, basic and diluted, for the same quarter in 2025. For the nine months ending September 30, 2025, the net loss totaled -$15.6 million.

Personnel costs for specialized scientific and management teams are embedded within the R&D and G&A figures. For instance, the third quarter of 2025 saw lower research and development personnel costs, which helped offset some of the R&D expense increase. The company appointed Gene Mack as President and Chief Executive Officer effective January 6, 2025, with Gianluca Fuggetta assuming the role of Senior Vice President Finance and Principal Financial Officer.

Intellectual property maintenance and patent prosecution fees are a necessary, though often less detailed, component of the operating expenses. While specific line items for these fees aren't explicitly broken out for Q3 2025, they contribute to the overall G&A spend. The company's focus on its Magellan™ drug discovery platform and advancing GT-02287 necessitates ongoing IP protection costs.

Here is a quick look at the operating expense trends for the third quarter:

Metric	Q3 2025 Amount (USD)	Q3 2024 Amount (USD)
Research and Development (R&D) Expenses	$2.8 million	$2.6 million
General and Administrative (G&A) Expenses	$1.9 million	$1.8 million

The cash position also reflects this cost structure. Cash and cash equivalents stood at $8.8 million as of September 30, 2025, down from $10.4 million as of December 31, 2024. The company did see $11.9 million in cash inflows from financing activities over the nine months ending September 30, 2025, to help fund operations.

You can see how the quarterly losses stack up:

• Net Loss for the three months ended September 30, 2025: -$5.3 million.
• Net Loss per Share for Q3 2025: ($0.15).
• Net Loss for the nine months ended September 30, 2025: -$15.6 million.
• Cash and Cash Equivalents as of September 30, 2025: $8.8 million.
• Cash and Cash Equivalents as of December 31, 2024: $10.4 million.

For context on earlier 2025 spending, here are the Q1 figures:

• R&D Expenses for Q1 2025: $2.26 million.
• G&A Expenses for Q1 2025: $2.11 million.
• Net Loss for Q1 2025: -$4.53 million.

Finance: draft 13-week cash view by Friday.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Gain Therapeutics, Inc. (GANX) as of late 2025, and honestly, it's what you'd expect for a clinical-stage biotech. The focus isn't on current sales, but on securing the capital needed to push the pipeline forward.

Minimal current revenue; consensus Q4 2025 revenue forecast is $0.000.

• As per analyst ratings, the consensus revenue forecast for 2025Q4 is $0.000.
• Analyst forecasts indicate that Gain Therapeutics' revenue in 2025 is projected to be $0.
• Trailing twelve months (ttm) revenue is listed as n/a.

Non-dilutive grant funding from foundations like The Michael J. Fox Foundation.

Gain Therapeutics, Inc. has secured non-dilutive funding, which directly offsets operating expenses like Research & Development (R&D). For instance, R&D expenses decreased in Q1 2025 partly due to research grant income.

• Funding support has been announced from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA for the GT-02287 program.
• The company was supported by Innosuisse, the Swiss Innovation Agency, with a grant of CHF 2.5 million (approximately $2.8 million) to develop GT-02287.
• This funding added to a prior CHF 1.5 million funding received from Innosuisse in 2021.
• The program also received support from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research.

Potential future milestone payments from strategic licensing agreements.

The business model anticipates revenue generation down the line from its proprietary platform and licensed assets. The terms of these arrangements typically outline specific non-cash or cash payments tied to progress.

Revenue Component Type	Typical Structure in Biotech Licensing	Specific Known Arrangement
Initiation Fee	Non-refundable fee upon contract signing.	The terms of collaboration and licensing agreements typically include a non-refundable initiation fee.
Milestone Payments	Payments upon achieving future development and regulatory goals.	Future development and regulatory milestone payments are a typical component.
Royalties	Percentage of net sales from a commercialized licensed product.	Royalties on net sales of the licensed product are a typical component.

Potential future royalties from commercialized products developed via the platform.

Should any of the pipeline programs, such as the lead candidate GT-02287, reach commercialization, royalties on net sales would become a significant, recurring revenue stream. The Magellan™ platform is used to identify targets for various therapeutic areas.

Equity financing through public offerings (ATM or secondary offerings).

This is the most concrete, near-term source of cash infusion to fund operations, given the lack of product sales. You've seen them execute on this recently to extend the cash runway.

• In November 2025, Gain Therapeutics, Inc. filed for an At-The-Market (ATM) offering authorization up to $35,530,980.56.
• As of November 2025, the company had already sold an aggregate of $14,469,019.44 under the original ATM agreement established in September 2024.
• In July 2025, the company priced an underwritten public offering expecting aggregate gross proceeds of approximately $7.0 million.
• For the second quarter of 2025, the company highlighted a public offering that raised $7.1 million.

The cash position as of September 30, 2025, was $8.8 million.