The meteoric rise of CRISPR, the genome-editing tool in the last decade, remains unmatched. CRISPR has been used in biological research, translational medicine, food technology, among many others. Its impact on the scientific community has been so monumental that it earned the Nobel Prize for its co-developers, Dr. Jennifer A. Doudna and Dr. Emmanuelle Charpentier.
Riding on this wave to capture the market with their proprietary CRISPR gene editing tools are several small to big companies. One such company is Spotlight Therapeutics Inc., an early-stage biotech that is based in California. In the ongoing BIO CEO & Investor Digital Conference, Dr. Mary Haak-Frendscho, the President & CEO, was interviewed on the clinical progress made by CRISPR-based therapeutic approaches.
Spotlight Therapeutics Inc.
Several ongoing and published studies have shown that CRISPR has the potential to alter genetic diseases in humans. Even though significant progress has been made to modify genomes by using nucleases, delivering the Cas/RNA complex to specific cells remains a huge challenge. Additionally, side effects, size limitations, among others can pose challenges, thus eliminating half the patients in need of therapy. Spotlight Therapeutics has decided to take on this problem in full frontal mode.
Dr. Haak-Frendscho introduced the new class of biologics called Targeted Active Gene Editing (TAGE) agents that her company is developing. Each of these TAGE agents contains a cell-targeting domain, a cell-penetrating peptide (CPP), and a Cas protein with a guide RNA. On entering the in vivo environment, these agents target immune cells to perform gene editing while reprogramming the tumor microenvironment and initiating an antitumor response.
When asked about the benefits of their delivery method, she mentioned that while traditional cell therapy has shown to cause viral inflammation in patients, their method can potentially avoid such issues and encourage higher levels of access in patients.
Pipeline and Partnerships
Spotlight Therapeutics is keen on developing therapies pertaining to immuno-oncology, regulating tumor microenvironment, among others. Their pipeline includes a potential candidate which will leverage the permanence of gene editing in oncology, aimed to reach clinical trials by the end of this year. They further intend to expand their portfolio by empirically selecting nucleases to fight other disease indications.
Clinical trials, in general, are expensive and need to be planned in a detailed manner, which is why partnerships with larger companies are sometimes essential as they have already been down this road. Besides oncology, Spotlight would like to strategically partner with companies with deep expertise and commitment to treating rare genetic diseases.
Competition and Challenges
Spotlight Therapeutics has already raised $30 million, which will last until 2022, and is expected to start its next fundraising late this year. Despite that, Dr. Haak-Frendscho is aware of the challenges of the rapidly evolving field and competition.
Multiple startups, as well as academic institutions, are investing in the development of engineered Cas-9. This has led to a complex Intellectual Property (IP) landscape. However, she is optimistic that Spotlight Therapeutics holds a key part to the puzzle in the field of CRISPR therapy and will be able to negotiate deals with major IP holders in the future.
NTrans Technologies, Ligandal, Intellia therapeutics, Genedit, and Poseida Therapeutics are some of the potential competitors of Spotlight. These companies are all working towards developing newer strategies to deliver CRISPR-based therapies in various diseases. With CRISPR therapy gaining a lot of attention both from the scientific world and the investors, it will be interesting to see if any of the multinational corporations take over or the start-up companies like Spotlight Therapeutics will dominate this space.
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