The first RNA therapeutic to treat Spinal Muscular Atrophy (SMA) was Spinraza, a drug developed by Cold Spring Harbor Laboratory (CSHL), Ionis, and Biogen. Inspired by Spinraza’s impact and driven by the desire to deploy AI/ML technologies to discover revolutionary RNA drug targets and treat human diseases, CSHL researchers Dr. Maria Luisa Pineda and Dr. Martin Akerman joined forces to create Envisagenics.
Envisagenics is built on the connection between RNA splicing errors and disease. SpliceCore®, Envisagenics’ proprietary AI/ML drug discovery platform, identifies novel tumor-specific immunotherapeutic epitopes. ENMEDIA spoke with Envisagenics co-founders Dr. Maria Luisa Pineda and Dr. Martin Akerman on all things AI, being a leader, and how to successfully transition between academia and industry.
ENMEDIA: What allowed you to successfully translate your technology from academia to biotech?
MARTIN: Envisagenics was spun out of Dr. Adrian Krainer’s lab at Cold Spring Harbor Laboratory (CSHL), where I completed my post-doctoral training and was part of the team that developed Spinraza® in collaboration with Ionis and Biogen — the first RNA therapeutic approved to treat Spinal Muscular Atrophy (SMA). Both my co-founder Maria, who had recently completed her PhD at CSHL and was working in venture at the time, and I were struck by the drug’s profound impact on the lives of children with this devastating disease. In 2014, we began to employ AI/ML in drug discovery with Envisagenics and extended our vision to treat other human diseases. Transitioning from academia to industry allowed us to widen the scope of our efforts. We were able to build our proprietary drug discovery platform, SpliceCore®, to discover and develop drugs for hundreds of diseases associated with RNA splicing errors. It became easier to match resources to promising R&D opportunities and generate meaningful results faster by focusing exclusively on discovering and validating new drug candidates.
In the early stages of the company, we were awarded several federal grants to help fund our research and eventually started partnering with biopharmaceutical companies like Bristol Myers Squibb to advance the development of our drug candidates. We also built a strong scientific and strategic advisory board to help us navigate the transition from academia to industry. Our passion for scientific principles and AI/ML combined with our industry-leading platform allows us to continue to drive innovation and efficiency in drug discovery — ultimately offering new treatment alternatives to patients faster than ever.
ENMEDIA: How is RNA drug discovery and Envisagenics changing the drug discovery landscape?
MARIA & MARTIN: In recent years, we have seen an abundance of new drug modalities emerge, such as RNA therapeutics and immunotherapies. This has created a high demand for new drug targets, as pre-existing targets were originally designed for small molecules and were not optimized for these newer modalities. While other companies focus on repurposing old drug targets with limited success in the clinic, Envisagenics focuses on discovering novel targets optimized for newer drug modalities.
Our target discovery platform, SpliceCore®, uses AI/ML to discover new drug targets that result from RNA splicing errors. RNA splicing is a molecular process that turns budding RNA molecules into messenger RNAs that carry coherent genetic messages. When splicing fails, it sends shockwaves of wrong genetic messages throughout the cell, causing cancer or neurodegeneration.
In cancer, Envisagenics’ platform uses splicing errors to single out cancer cells and train the immune system to attack them. In neurodegeneration, we use RNA therapeutics to correct splicing errors and re-establish normal gene expression.
We have spent the last decade cataloging and annotating a proprietary map of millions of RNA splicing events that enable the discovery of novel disease-specific splicing errors that can be targeted by antibodies or modulated by RNA therapeutics.
Given this enormous search space, SpliceCore is 400 times more likely to find novel drug targets than traditional genomic methods — opening a vast universe of potential drug candidates to treat patients with 370 diseases associated with splicing errors.
By concentrating on RNA splicing, we can stratify patients with more precision, yielding drugs more likely to have clinical success while reducing these endeavors’ overall time and cost.
ENMEDIA: How does Envisagenics influence or take advantage of the innovation ecosystem?
MARIA: Envisagenics is a biotech company at the forefront of AI/ML and RNA splicing research. Not only are we leading the effort to incorporate AI/ML in drug discovery, but we are also changing the face of the biotech industry with an unprecedented approach to drug discovery. As a woman- and minority-led business, we have built a diverse and inclusive organization with a mission-driven team of innovative thinkers. We actively promote diversity in the industry, particularly encouraging the next generation of scientists through various STEM programs. As an example, we participate in the NYC LifeSci Internship program to provide mentorship and exposure to young professionals in entrepreneurship, biological sciences, bioinformatics, data analytics and computer engineering. We have been lucky to retain many interns as full-time employees, further fostering our unique corporate culture.
Envisagenics is also one of the founding members of the Alliance for Artificial Intelligence in Healthcare (AAIH). I have served as Vice Chair and Secretary on the executive board for the past three years. The AAIH is a non-profit coalition of academic institutions, pharmaceutical companies, and research organizations with a shared goal to realize the full potential of AI in healthcare. Together, we encourage collaboration and drive the adoption of responsible AI in all aspects of the biopharma industry.
At Envisagenics, we believe people and collaboration drive innovation. To leverage seismic shifts in the biotech industry, such as AI, we need to work together to develop standards that consider ethical implications while fostering technological advancement. Together with AAIH, Envisagenics is reshaping the industry, working to save patients’ lives everywhere with innovative technologies that deliver cutting-edge therapies in record time.