2020 will soon be a record year for biotechnology. In the first half of 2020, the biotechnology industry obtained $16.55 billion in investment. Cell and gene therapy biotechs secured the lion share of investment by raising $10.7 billion. Notably, the amount raised by cell and gene therapy biotechs increased by 120% during the same time period of 2019.1 Thus far, the increase in this year’s investing appears to be driven by venture capitalists (VCs) as they contributed approximately 30% of the total investment.
Classical gene therapy involves the use of a recombinant form of a virus to deliver genes or gene-modifying nucleic acids to target tissues in order to replace defective genes causing disease. Genetically modified cells are made from patient-derived stem or allogeneic cells, and a viral vector that mediates the integration of genes that will enable the cells, when implanted back into the patient, to propagate and replace the diseased cells and enable normal tissue function. Both approaches are widely accepted as having the potential to change the way we treat congenital, degenerative, and chronic diseases.
Since the first gene therapy trials in the early 1990s, the road has been long and troubled with authorizing this approach for clinical use. However, recent successes have shown promises with the approvals of Glybera and Imlygic, viral vectors designed for gene therapy of lipoprotein lipase deficiency and melanoma, respectively. In addition, Kymriah and Yescarta, two forms of T-cells that are removed from a patient and genetically engineered to make specific cell surface receptors and then transferred back into patients, have been approved for B-cell acute lymphoblastic leukemia and non-Hodgkin’s lymphoma, respectively. Thus, gene and cell therapies have finally arrived and appear they will only become better through the integration of important enabling technologies. Moreover, it appears VCs have identified and are pursuing the companies that are creating these enhancement technologies.
Gene and Cell Companies with the Highest Investments from Venture Capitalists
Sana Biotechnology appears to have cracked the code for the development of “universal donor cells”. Although the concept of cell replacement therapy is outstanding, the process of generating pluripotent stem cells is too long and expensive and the scientific process is highly variable with regards to the quality of the cells generated. In addition, there has yet to be a reliable scientific approach to make replacement donor cells non-immunogenic. Sana Biotechnology announced in 2019 that it had the ability to not only genetically reprogram cells, but make them scalable, cost-efficient and non-immunogenic. 3
Sana Biotechnology reported in June 2020 that its initial series A funding raised over $700 million from multiple investors including VCs, including ” ARCH Venture Partners, Flagship Pioneering, Canada Pension Plan Investment Board, Baillie Gifford, F-Prime Capital, Alaska Permanent Fund, the Public Sector Pension Investment Board, Bezos Expeditions, GV, Omega Funds, Altitude Life Science Ventures.” 4 With this initial funding, Sana Biotechnology hopes to continue developing gene delivery, immunology, stem cell biology, and gene modification and control.4 The amount that Sana Biotechnology raised is almost as much “as much as the next five largest private rounds raised by Orca Bio, Elevate Bio, Legend, Freeline Therapeutics, and Poseida.”5 Comparatively, the largest amount invested in a biotechnology company who is not researching cell and gene therapy is $390 million, in Series D funding raised by Grail who are focusing on blood tests for cancer diagnosis.6
Lyell Immunopharma is a cell therapy company that is focused on “advancing the science of T cell differentiation, functionality, and target specificity in order to develop curative treatments for solid tumors. These technologies can be used as a platform for multiple new cell therapies that can be applied across a broad range of cancers”7. In total, the company raised $493 million in Series C funding with an undisclosed amount of investors. Pharmaceutical firm GlaxoSmithKline, which has a capital venture fund under its name, Action Potential Venture Capital, reported that they invested £258 million (approximately $320 million) on equity investments primarily into Lyell Immunopharma.8 Lyell Immunopharma raised more money in Series C funding than any other biotechnology startup in the series.9 The second largest was XtalPi, an AI drug startup, with $318.8 million. This is roughly $175 million less than Lyell Immunopharma. The amount that SoftBank, the lead investor for XtalPi, invested in the company is undisclosed at this time. However, the data signifies that it was less than $318 million as XtalPi had seven investors participate in Series C funding, four of them being the main contributors.9
Orca Bio is a biotechnology company that focuses on developing high precision allogeneic cell therapies. These cell therapies are “designed to safely and effectively replace a patient’s blood and immune system with a healthy one. The manufacturing platform sorts donor blood with single-cell precision and a high level of purity and speed, enabling the creation of proprietary, optimal therapeutic mixtures of immune and stem cells that have the potential to transform allogeneic cell therapy.”10 The company raised in Series D funding $192 million, in addition to the already roughly $200 million that was raised in previous years. Their lead investor was LightSpeed Venture Partners, along with one other undisclosed investor. Other investors in this series included “8VC, DCVC Bio, ND Capital, Mubadala Investment Company, Kaiser Foundation Hospitals, Kaiser Permanente Group Trust, and IMRF.”10
Interestingly, on September 02, 2020, Orca Bio and Lyell Immunopharma announced a research partnership to “jointly identify next-generation T-cell therapies that will combine the high-precision purification T cell technologies from Orca Bio with the scientific expertise in T cell biology from Lyell to generate potentially synergistic therapeutic solutions for solid tumors.”11 This indicates that there will be more than $600 million in VC funding between the two biotechnology startups from their respective latest funding rounds.
Between all the investments made in between Series A to D, Sana Biotechnology and Lyell Immunopharma rank number two and number three, respectively, in the largest amount of money raised during this past year.12 Just between the two companies, they raised $1.193 billion out of the $10.7 billion that was invested in cell and gene therapy. This accounts for roughly 40% of all the money invested by VCs in cell and gene therapy.
The funding of biotechnology startups who are studying gene and cell therapy is not surprising. The field made some significant advancements in 2019 and is forecasted to grow over 24% CAGR (Compound Annual Growth Rate) over 2018-202413. The revenue in this field is expected to increase over 30.9% between 2019-2025.13 Hence, cell and gene therapy are now becoming a clinical reality and the aggressive investment is a strong signal that this form of therapy will impact the clinic and business in the near future.
Cell and gene therapy had a successful 2020. The amount of funding and the multitude of investors funding each company shows that this area of research is what the world, as well as the investors, are looking for. The funding will help these companies reach the world market at a faster pace, thus advancing science and humanity.
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Author: Nicole Ludwiak
- Alliance for Regenerative Medicine. (2020, August 6). New Report: Regenerative Medicine & Advanced Therapies Sector Thriving Despite COVID-19. GlobeNewswire News Room. http://www.globenewswire.com/news-release/2020/08/06/2074281/0/en/New-Report-Regenerative-Medicine-Advanced-Therapies-Sector-Thriving-Despite-COVID-19.html
- Constance, J. (2020, September 3). Small Biopharma Companies Emerging in Gene and Cell Therapy. BioSpace. https://www.biospace.com/article/small-biopharma-companies-emerging-in-gene-and-cell-therapy-/
- January 04, 2019 Sana Biotechnology Press Release. https://sana.com/sana-biotechnology-launches-focusing-on-engineering-cells-to-create-a-new-class-of-medicines-4/
- (2020, June 23). Announces Completion of Initial Financing. Sana Biotechnology. https://sana.com/sana-biotechnology-announces-completion-of-initial-financing/
- Idrus, A. A. (2020, August 6). In the face of COVID-19, cell and gene therapy space shows ‘remarkable resilience:’ report. FierceBiotech. https://www.fiercebiotech.com/biotech/face-covid-19-cell-and-gene-therapy-space-shows-remarkable-resilience-report
- Glasner, J. (2020, July 22). Life Science Funding Spikes In…Crunchbase News. https://news.crunchbase.com/news/life-science-funding-spikes-in-2020/
- Bio, O. (2020, September 2). Orca Bio and Lyell Immunopharma Announce Research Partnership. GlobeNewswire News Room. https://www.globenewswire.com/news-release/2020/09/02/2087609/0/en/Orca-Bio-and-Lyell-Immunopharma-Announce-Research-Partnership.html
- Lavine, R. (2020, March 30). GSK displays Lyell Immunopharma investment. Global Corporate Venturing. https://globalcorporateventuring.com/gsk-lyell-immunopharma-investment/
- Advanced Search | Funding Rounds. (, 2020). Crunchbase. https://www.crunchbase.com/discover/funding_rounds/4a2ad41015d5f0a14db0b701ed690baf
- Bio, O. (2020a, June 17). Orca Bio Emerges With Nearly $300 Million to Transform Allogeneic Cell Therapy. GlobeNewswire News Room. https://www.globenewswire.com/news-release/2020/06/17/2049369/0/en/Orca-Bio-Emerges-With-Nearly-300-Million-to-Transform-Allogeneic-Cell-Therapy.html
- Orca Bio and Lyell Immunopharma Announce Research Partnership. (, 2020). Orca Bio. https://orcabio.com/2020-09-02/
- Advanced Search | Funding Rounds- Series A to Series D. (2020). Crunchbase. https://www.crunchbase.com/discover/funding_rounds/ad86f5e2a56b8b304ad48489fe3e1126
- Cell & Gene Therapy Market – Global Outlook and Forecast 2020-2025. (, 2020). Report Linker. https://www.reportlinker.com/p05827567/Cell-and-Gene-Therapy-Market-Global-Outlook-and-Forecast.html?utm_source=PRN