Spotlight on Biotech Companies
Spotlight on Biotech Companies Pursuing In-Vivo Gene Editing Through Novel Platforms
The spotlight is on biotech companies pursuing in vivo gene editing as the way forward for next-gen gene therapies. With clinical data released in June showing promising results of the first in vivo CRISPR-based gene therapy, investor interest is also soaring, with investment up to near $10B after clinical trial results have shown no serious adverse effects, and a high response to treatment (Issi, 2021). Following, we highlight a few companies working on in vivo gene editing through novel platforms.
GenEdit, co-founded in 2016 by UC Berkeley graduate Kunwoo Lee, started out as a small San Francisco-based biotech company the same year Lee graduated from Berkeley. Having worked in Jennifer Doudna’s lab at UC Berkeley to complete his post-doc, and inspired by Doudna’s work with CRISPR-Cas9, Lee set out to improve on the drawbacks of gene editing. One of these drawbacks is what Lee calls the “delivery problem” (DeFeudis, 2021). Most gene therapies are currently delivered using AAV vectors. The use of AAV vectors means there is a high chance of the gene therapy being ineffective, as many patients have a natural immunity to these delivery vehicles, and re-dosing is virtually impossible.
The biotech has just closed a $26M Series A funding round, which includes Eli Lilly as well as DCVC Bio, Sequoia Capital, Korea Investment Partners, and nearly a dozen others (LaHucik, 2021).
GenEdit plans to use the funds to further develop its NanoGalaxy Platform, which produces non-viral, chemically made polymer nanoparticles that are capable of delivering multiple doses of gene therapy in vivo, without triggering an immune response. The company’s sophisticated platform selectively targets different tissue and cell types, and has the ability to deliver RNA, DNA, or CRISPR proteins. One of the benefits of being able to re-dose is that smaller initial doses may be delivered to patients to avoid unwanted side effects of a single larger dose. The particles are also able to carry more therapeutic material, and cost less to be manufactured than standard delivery systems (DeFeudis, 2021).
There are other biotech companies pursuing in vivo gene editing. Code Biotherapeutics (a Philadelphia-area biotech), is developing a platform called 3DNA. The platform enables the delivery of large genes, improves cellular targeting, reduces immunogenicity, enables re-doseability, and simplifies manufacturing. Because it is not limited by size, the platform enables the delivery of gene therapies across multiple tissues and organs. Code’s current focus is on Duchenne muscular dystrophy and type 1 diabetes, and closed a seed funding of $10M in April, backed by several VCs including Takeda Ventures, Inc.
Generation Bio is on a mission to create a new class of non-viral gene therapy that is re-doseable, offers long-term efficacy, and works across a range of rare and prevalent diseases. The biotech closed a $230M IPO last June to fund its preclinical push for gene therapy treatment for diseases of the liver and retina (DeFeudis, 2021).
The company’s platform uses closed-ended DNA (ceDNA), which is capable of treating genetic diseases that involve larger genes, and can even carry multiple genes to a cell in one therapeutic dose. ceDNA also has durable expression, meaning one treatment is capable of lasting years. The ceDNA is carried into the cell’s nucleus via a cell-targeted lipid nanoparticle (ctLNP), which eliminates the risk of immunogenicity or activation of the immune system and allows for re-dosing if necessary. The platform also uses enzymatic manufacturing, which allows for rapid scaleability of consistent, highly pure ceDNA.
Bioengineering advancements and AI have enabled the creation of next-generation gene therapy platforms for biotech companies pursuing in vivo gene editing. With funding at an all time high for companies that fit the bill, there is ample opportunity for life sciences executive search consultants to place the talented researchers, developers, medical, and business executives to run these cutting-edge companies. Talented individuals are needed in these positions to synthesize the science and technology for necessary improvements in gene therapy, and to secure the financial backing required that will fund the advancement of pipelines.
If you’re interested in learning more about BayBridge’s Talent Management approach to executive search, please get in touch with one of our expert life sciences consultants today.
References
DeFeudis, N. (23 September 2021). Eli Lilly gets behind latest approach to solving gene therapy’s delivery problem. Endpoints News. https://endpts.com/eli-lilly-gets-behind-the-latest-approach-to-solving-gene-therapys-delivery-problem/
LaHucik, K. (23 September 2021). GenEdit strikes Eli Lilly gold with $26M for gene therapy delivery system designed to fight side effects. Fierce Biotech. https://www.fiercebiotech.com/biotech/genedit-strikes-eli-lilly-gold-26m-for-gold-nanoparticles-gene-therapy-delivery-system
Issi, L. (20 September 2021). What’s driving record capital in genetic medicine? Fierce Biotech. https://www.fiercebiotech.com/sponsored/what-s-driving-record-capital-genetic-medicine
