Biotech investments have rolled up and down over the past few years, but companies developing innovative platforms with a revolutionary bent have a leg up, according to an analysis by McKinsey & Company. Investors are funneling money into companies focused on cutting-edge science like gene editing, cell therapies and machine learning. And in a crowded sector, having a headline-making story to tell doesn’t hurt, either.
From resurrecting extinct species to using CRISPR gene editing in animal-to-human organ transplants, these companies are making futuristic technologies, products and platforms for real-life science.
Ginkgo Bioworks’ cell programming tech is a hot commodity for biopharma partners
What sets it apart: Billing itself as “the organism company,” Ginkgo Bioworks offers its cell programming technology platform and capabilities to partners in industries like biotech and agriculture. In pharma, the company has worked with Merck & Co. to engineer biocatalytic enzymes for potential use in drug manufacturing processes, and with Prokarium to engineer bacteria for cancer treatments.
What’s new: Ginkgo Bioworks recently acquired AgBiome’s platform assets, which include its development pipeline, as well as more than over 115,000 fully sequenced and isolated microbial strains and more than 500 million unique gene sequences. The company also recently expanded their R&D partnership with Novo Nordisk to improve manufacturing of Novo’s medicines for “serious chronic diseases, including diabetes and obesity medications.”
What’s coming: Ginkgo Bioworks has many irons in the fire including one of its most recently announced projects: building an open-access, AI-enabled measles forecasting model with Northeastern University researchers. The work will be supported by a newly awarded grant from the Bill & Melinda Gates Foundation.
eGenesis uses CRISPR to make animal-human transplants possible
What sets it apart: The FDA made a landmark approval of the first CRISPR drug last year, and this milestone for the gene editing technology is one of many that could be on the way.
eGenesis is leveraging CRISPR to edit animal organs for human transplants. According to eGenesis, more than 100,000 people in the U.S. need an organ transplant, and every day, 20 people die waiting for one. eGenesis is working to develop human-compatible, genetically engineered organs.
What’s new: eGenesis made history in March when, for the first time, a genetically engineered pig kidney was transplanted into a living human recipient. The key to the transplant’s success were three classes of gene edits that prevent rejection. The eGenesis kidney used for the transplant, EGEN-2784, is the company’s lead product candidate for kidney transplant and was authorized by the FDA under the expanded access pathway for the end-stage renal disease patient who had no other therapeutic options. The patient was discharged at the beginning of April.
What’s coming: The eGenesis pipeline comprises candidates for heart, kidney and liver human-compatible organs, and it’s making clinical strides in showing their viability. In addition to the successful kidney transplantation, the company announced a major milestone in its liver program. In a partnership with Penn Medicine, eGenesis showed it was possible to circulate blood through a person using a genetically engineered pig liver outside the human body combined with an extra corporeal liver cross-circulation device made by the company OrganOx. The solution could provide a much-needed bridge for liver failure patients waiting for a transplant. They conducted the study on a recently deceased patient
Colossal Biosciences pushes the boundaries of gene editing
What sets it apart: This gene editing, “de-extinction” company aims to “resurrect” the woolly mammoth, among other species. By creating what Colossal CEO and co-founder Ben Lamm described to PharmaVoice as “proxy species,” the company could reintroduce eradicated species back into the wild and fill “ecological voids that were left in their absence.”
But there’s more to the company than this singular goal. After it builds its capabilities, Colossal plans to become a springboard for other spinoffs, such as Form Bio, a computational life sciences platform focused on speeding drug development that launched in 2022.
What’s new: In March, Colossal announced it was able to reprogram elephant iPSC cells, which had never been achieved before. iPSC, or induced pluripotent stem cells, can propagate indefinitely and grow into any cell type, such as heart or lung cells. According to the company, this breakthrough showed they could grow elephant sperm and egg cells for gestation, and eventually, edit them into mammoth cells for the same purpose.
What’s coming: In addition to “bringing back” the woolly mammoth, Colossal’s work could be applied to a host of other ecological and human health problems, from boosting endangered animal species, to enabling applications like plastic-eating bacteria, sustainable agriculture and disease remodeling.
Spiderwort’s regenerative medicine tech uses plant-based biomaterials
What sets it apart: Spiderwort is developing plant-based biomaterials that can repair and regenerate living tissue, acting as the “scaffolding” for regenerative medicine, including treating spinal cord injuries and soft tissue regeneration.
What’s new: Spiderwort was accepted last year into the MaRS Health Science Portfolio, which supports socially impactful health companies and institutions as they bring products to market.
What’s coming: Spiderwort has two products in development: CelluBridge, for the repair and regeneration of the spinal cord, which received a breakthrough device designation from the FDA, and CelluJuve, a cellulose-based dermal filler for injury recovery and cosmetic enhancement.