Insilico and GenEditBio are at the forefront of this cutting edge industry that is moving at lightning speed. On the ground, they’re having tremendous impact in addressing the injustice of rare diseases. Insilico, led by CEO Alex Aliper, is focused on creating “pharmaceutical superintelligence” with the intent of increasing therapeutic options for personalized patient treatment within the next 10 to 20 years. GenEditBio, co-founded by Tian Zhu, is taking their shot to do gene editing differently. The company is using innovative delivery systems to increase the efficacy of their treatments for rare genetic disorders.
With such cutting-edge AI technology, Insilico is revolutionizing the process of drug discovery. Humanetics produces digital twins of humans for use in virtual clinical trials. Aliper refers to this new procedure as “still in nascence.” This new technology enables researchers to more accurately simulate human responses to therapies without the costly and time-consuming development of traditional clinical trials. By utilizing automated laboratories that generate multi-layer biological data from disease samples at scale, Insilico can operate without human intervention, thereby expediting the research process.
All of the data created from these automated systems fuels into Insilico’s AI-driven discovery platform. This platform enables the company to train its models and collaborate effectively with external partners, enhancing the potential for breakthrough therapies. Aliper states, “We still need more ground truth data coming from patients,” highlighting the ongoing requirement for real-world data to validate their findings and models.
The mission of Gene Editing Bio perfectly aligns with Insilico’s work to develop editing and delivery mechanisms. Through its collaborative academic research the company has generated an extensive library of thousands of unique, nonviral, nonlipid polymer nanoparticles. These nanoparticles are specifically engineered to carry gene-editing tools right into the targeted cells. This level of precision is the hallmark of successful in vivo gene editing.
Tian Zhu points out that “efficient, tissue-specific delivery is a prerequisite for in vivo gene editing.” GenEditBio is on a mission to make gene editing as easy as word processing. It allows the treatments to be administered with one injection right into the damaged tissues. This new approach does more than improve accuracy—it has the potential to deliver more cost-effective and widely available treatments.
In today’s advanced biotech, we have the technology and capability to edit genes and design drugs, Zhu adds, yet thousands of rare diseases remain untreated. This announcement highlights the acute need for innovation in drug development and delivery systems.
It is GenEditBio’s testing methodology that really sets it apart from typical approaches. Instead of testing a single delivery nanoparticle, the company conducts simultaneous tests on thousands of particle variants. This approach speeds the discovery of the best and most effective delivery system and dovetails beautifully with the emerging focus on precision medicine.
At the same time as these companies pioneer the frontiers of biotechnology, they face some of the industry’s most pressing challenges. Aliper notes that the pharmaceutical industry is currently experiencing stagnation, stating, “We’re in a plateau of around 50 drugs [approved by the FDA] every year annually, and we need to see growth.” He emphasizes the need for innovative technologies to bolster productivity within the industry and address the labor and talent shortages that hinder progress.
AI-enabled drug discovery and CRISPR/Gene-editing technologies have cut their costs. This new direction holds great potential to change how we treat rare diseases. These firms use artificial intelligence, digital twins, and ninja delivery systems. Their mission is to improve upon currently available treatments and develop new treatments for disease areas that have been historically overlooked.