Aricle by Demaris Mills is president at Integrated DNA Technologies, a global genomics solutions provider, on fobes.com
The past seven years have brought a remarkable boom in genomics medicine. The first CAR-T cell therapy for cancer—genetically modified to recognize individual patients’ cancers—was approved by the U.S. Food and Drug Administration in 2017, with five additional CAR-Ts approved since. The first mRNA vaccines, developed in record time to treat Covid-19, hit the market in 2021. Seven gene therapies to correct inherited diseases have been approved, and in December 2023, the FDA green-lighted the first CRISPR gene-editing therapy to treat sickle cell disease.
It's truly exciting to imagine what the next generation of genomics medicines will offer for patients. A staggering 2,093 CAR-Ts, gene therapies and RNA medicines are in biopharmaceutical pipelines around the world, targeting a wide range of cancers, rare diseases, neurological disorders and more.
But the developers of these therapies are facing significant challenges. One of the toughest hurdles from a scientific perspective is guaranteeing the technological safety of gene and cell therapies for patients. These technologies are so new that every novel therapy undergoes a complex and often lengthy regulatory review process. Concerns for patient safety are top of mind for regulators, further slowing the path to market for new genomic innovations.
The solution? For the biopharma industry to match the pace of genomic innovations and speed new therapies to market, it will need to embrace innovative collaborations.
Streamlining Development With Platform Technologies
As one example, my company, Integrated DNA Technologies (IDT), a Danaher operating company, is a prominent player in a collaboration designed to accelerate the development of genomics therapies. In January 2024, Danaher teamed up with the Innovative Genomics Institute (IGI) to launch the Danaher-IGI Beacon for CRISPR Cures. The Beacon’s initial focus will be developing cell therapies for rare genetic disorders known as "inborn errors of immunity" (IEIs). We’re starting with two such diseases, but the goal is to build platform technologies that are expandable to the development of CRISPR treatments for any of the 500 or so IEIs.
The Beacon lines up well with the FDA’s recent focus on streamlining the development of genomics medicines. In May 2024, the agency unveiled a draft guidance on its Platform Technology Designation Program for Drug Development Guidance for Industry, which will provide a pathway for developers to use approved delivery vectors and gene-editing technologies for multiple products. This could lessen the need for developers to repeat the full (pre-)clinical research and development phase to demonstrate safety and efficacy, potentially speeding up the approval process.
Another good example of collaborative platform development was announced in April 2024 by Regeneron Pharmaceuticals and Mammoth Biosciences. They’re working together to create CRISPR therapies that can be delivered not just to the liver, where most gene-editing treatments are targeted today, but to many other tissues of the body. Regeneron is using antibodies to perfect the delivery of CRISPR via next-generation viral vectors, and Mammoth is making “ultracompact” gene-editing technology. By pooling their respective expertise, they hope to overcome challenges in delivering CRISPR therapeutics into the body, which ultimately could increase the number of diseases that can be addressed with gene editing—and the population of patients who could benefit from them.
Focus On Safety
Collaborations could also help genomics innovators improve the safety of cell and gene therapies. One priority in the industry is to develop alternatives to the viral vectors that are used to deliver some genomics therapies, as they can cause immune reactions and other side effects. With CRISPR, there’s a risk of “off-target” effects, in which the enzyme used to edit the genome can potentially affect other genes and cause adverse effects. Several alternatives to the commonly used Cas9 enzyme are being investigated for clinical use, so there’s still more work to be done to guarantee the safety of CRISPR therapies.
Although collaborations have long been common in the industry, the rise of genomics medicine may require some out-of-the-box approaches. Drug makers may need to partner more with device makers and vice versa, and all of us in the biopharma industry should double down on our academic collaborations, too. It’s the only way we can ensure the thousands of genomics innovations in the pipeline make their way to the patients who so desperately need them.