Revolutionizing Treatment: Gain Therapeutics Leads the Way in AI-Driven Drug Discovery for Complex Diseases


What Makes a Truly Valuable AI Drug Discovery Platform?

The value in AI drug discovery platforms lies in their ability to target known, yet previously unreachable, therapeutic targets. Gain Therapeutics’ Magellan™  exemplifies this by combining AI with a physics-based approach to identify novel allosteric binding sites on disease-implicated proteins. This method, focusing on traditionally ‘undruggable’ targets without requiring extensive pre-existing data, sets it apart from other AI-based platforms. The entry of compounds developed through this platform into clinical trials is a significant milestone, showcasing its potential in drug discovery.

A Closer Look at GT-02287 Reveals a Potential Best-In-Class GCase/GBA1 Therapeutic for Parkinson’s Disease

Gain’s drug GT-02287 shows promise in treating Parkinson’s Disease, particularly in cases involving GBA1 mutations. Its mechanism of action – preventing the misfolding of GCase in the endoplasmic reticulum (ER) – is a significant advancement. This is in contrast to other drugs that focus on increasing GCase activity but may not address the underlying issue of misfolded GCase. GT-02287’s ability to stabilize GCase, ensuring its proper function in the lysosome and addressing various GBA1 mutations, highlights its potential as a superior therapeutic option.

Gain’s A1AT Deficiency Asset

In addition to Parkinson’s disease, Gain Therapeutics is exploring treatments for alpha-1 antitrypsin (A1AT) deficiency. This research, though in early stages, has received a €1.2 million grant for developing small molecule allosteric regulators. A1AT deficiency primarily affects the lungs, leading to severe COPD-like symptoms, and can also cause liver issues. Current treatments focus on symptom management but a cure remains elusive. Gain’s approach in this area is believed to be unique and potentially more effective.

AI in Drug Discovery: Navigating the Challenges in Complex Disease Treatment

The integration of AI and machine learning into drug discovery and development has been a growing trend in recent years. Yet, the past 18 months have delivered a reality check. Various molecules developed using AI have not succeeded in clinical trials, being either deprioritized or failing to achieve expected outcomes. This includes a notable AI-developed drug for dermatitis which did not produce the desired results and another for obsessive-compulsive disorder (OCD) which faced similar challenges. Such instances have put the spotlight on the limitations and complexities involved in AI drug discovery, especially when addressing intricate and multifaceted diseases like anxiety disorders and OCD. These outcomes have served as a reminder of the hurdles that still need to be overcome in this innovative field.


The journey of AI in drug discovery, despite facing challenges, shows a promising future with platforms like Gain Therapeutics’ Magellan™  This platform’s ability to identify new therapeutic targets and its success in bringing compounds to clinical trials highlights the potential of AI in revolutionising drug discovery. GT-02287 in particular stands out as a potential best-in-class therapy for Parkinson’s Disease thanks to its unique approach in addressing the root cause of GCase misfolding. Additionally, Gain’s foray into developing treatments for A1AT deficiency demonstrates the company’s commitment to tackling complex diseases with innovative approaches. As the field of AI-driven drug discovery evolves, it holds the promise of delivering more effective and targeted treatments for various diseases.