Finance Watch: Cell And Gene Therapy Firms Raised Nearly $20bn In 2020

Private Company Edition: Venture capital mega-rounds continue a brisk pace with machine learning-based drug discovery firm insitro and gene therapy developer Graphite Bio among recent $100m-plus deals. Also, Argobio raised €50m to fund five start-ups in Europe. 

Cash is flooding into biopharmaceuticals, but regenerative medicine companies in particular have raked in large sums of cash – in part, because of the high upfront investment in manufacturing, but also because of the potential to cure intractable diseases. In 2020, that meant a record-breaking $19.9bn was invested in cell and gene therapy developers globally – double the $9.8bn raised in 2019 and substantially higher than the prior record of $13.5bn in 2018 – according to a new report from the Alliance for Regenerative Medicine (ARM).

Investment in cell and gene therapy firms last year exceeded the 2018 total before year-end with $15.9bn raised as of the third quarter of 2020. (Also see "Finance Watch: Olema IPO Raises $209m To Advance OPI-1250 For Breast Cancer" - Scrip, 20 Nov, 2020.) And for privately held regenerative medicine companies, 2021 appears to be off to a strong start as well with many $100m-plus venture capital mega-rounds, including a $525m series C round for cell and gene therapy manufacturing hub and company creator ElevateBio, LLC, announced on 15 March.

Next-generation gene editing specialist Graphite Bio also announced a mega-round on 15 March with a $150m series B financing, for a total of $195m in funding to date, including a $45m series A round in September. (Also see "Finance Watch: Two New VC Funds Raise $755m To Fund Innovation" - Scrip, 23 Sep, 2020.) (See table below for more on this and other recent venture capital deals.)

The ARM report released on 16 March noted that the $19.9bn invested in regenerative medicine companies in 2020 included $5.6bn in venture capital investment. Cell and gene therapy and gene-editing firms also raised $3.7bn in initial public offerings, $6bn from follow-on offerings and $1.2bn from private placements. Upfront fees from partnering deals totaled $3bn last year.

Among the largest VC deals for cell and gene therapy companies in 2020, Sana Biotechnology, Inc. closed a $700m funding round in June. (Also see "Finance Watch: Sana Reveals Cash Haul Of $700m In Under Two Years" - Scrip, 23 Jun, 2020.) Sana went public in the US this year, grossing $587.5m in its IPO in February. (Also see "Finance Watch: Ten IPOs In Three Days Raise $1.9bn" - Scrip, 8 Feb, 2021.)

Mega-Rounds Grab Headlines, But €50m Goes Far For Start-Ups

Deals In Depth

For private company financings and start-up news in depth, also see:

Amunix Raises $117m To Take Novel T-Cell Engager Into The Clinic

Alvotech Completes $100m Private Placement

AgomAb Identifies Regenerative Pathway Modulators, Attracts $74m Investment

Arix Launches Twelve Bio With Aim To Improve On CRISPR Gene-Editing Tech

 

Flashy venture capital mega-rounds to fuel big growth are headline grabbers, but a lot of investors are also providing smaller amounts of funding and large amounts of expertise to help start-ups reach early research milestones. 

That is a niche that Argobio SAS aims to help fill. The newly created incubator announced on 2 March that it has €50m ($59.9m) to fund at least five companies with innovative programs sourced from European academic institutions.

Paris-based Argobio was initiated by the health care VC firm Kurma Partners and Bpifrance, the French national investment bank, which partnered with strategic investors – the private pharmaceuticals company Angelini Pharma, the drug discovery firm Evotec SE and the biomedical research center Institut Pasteur. They will fund start-ups working in select therapeutic areas, including rare diseases, neurological disorders, oncology and immunology.

Argobio will identify academic programs and incubate these ideas in newly formed companies that will be advised by the incubator’s in-house experts, including Yves Ribeill, the founder and former chairman and CEO of Scynexis, Inc.; former Wellcome plc executive and serial biotech company founder and executive Neill Mackenzie; and Adocia co-founder Rémi Soula. Kurma managing partner Thierry Laugel will serve as president of Argobio, and Bpifrance director of biotech and ecotech investment Laurent Arthaud will chair the supervisory board.

Recent Venture Capital Financings
Company	Investors	Use Of Proceeds
insitro; South San Francisco	Just 10 months after insitro raised a $143m series B round (and less than two years after its $100m series A financing), the company said on 15 March that it closed a $400m series C round led by Canada Pension Plan Investment Board (CPPI Investments), a series B investor. (Also see "Finance Watch: VC Mega-Rounds For Machine Learning, CNS Disorders, Academic Discoveries" - Scrip, 1 Jun, 2020.) Current investors Andreessen Horowitz, funds and accounts advised by T. Rowe Price Associates Inc., Casdin Capital, and funds and accounts managed by BlackRock also participated along with ARCH Venture Partners, Foresite Capital, GV, Third Rock Ventures, Two Sigma Ventures, HOF Capital, Alexandria Venture Investments, Temasek, Softbank Investment Advisors, an undisclosed global investment group, and an undisclosed US payer provider health system.	The company noted that in 2020 it built out its machine learning-driven target discovery platform, made progress under its 2019 collaboration with Gilead Sciences, Inc. in non-alcoholic steatohepatitis (NASH), entered into a partnership with Bristol Myers Squibb Company in amyotrophic lateral sclerosis (ALS), acquired Haystack Sciences to move from target discovery to drug development, and recruited former Merck Research Laboratories president Roger Perlmutter as a member of its board of directors. (Also see "Deal Watch: Bristol Teams Up With Insitro, Sensyne In AI-Directed Collaborations" - Scrip, 30 Oct, 2020.) The series C financing, combined with insitro’s series B cash, will fund the company’s ambition to deliver drugs to patients faster and with fewer clinical trial failures. Insitro will expand its technology platform capabilities and its R&D pipeline, seek complementary technologies and in-license drug candidates that it can validate with its machine learning platform.
Valo Health LLC; Boston	Valo, the Flagship Pioneering-created start-up that disclosed a $190m series B round in January, said on 9 March that the round has grown to $300m with an additional $110m investment from Koch Disruptive Technologies. The company said it has now raised more than $450m to date. (Also see "Finance Watch: Biopharma VC Deals Hit New Record Of $27.4bn In 2020" - Scrip, 18 Jan, 2021.)	The company’s Opal Computational Platform combines machine learning with patient data to discover and develop first-in-class and best-in-class therapies across a variety of diseases, but with an initial focus on cancer, neurodegenerative and cardiovascular diseases. Valo aims to reduce the cost, time and failure rates associated with drug development. (Also see "Finance Watch: Big VC Money Backs Tech-Enabled Drug Discovery" - Scrip, 2 Oct, 2020.)
Vaccitech Limited; Oxford, UK	The company said on 17 March that it raised $168m in series B financing, including $43m in previously issued convertible loan notes that converted to series B shares. M&G Investment Management (part of M&G plc) led the round with participation from new investors Tencent, Gilead, Monaco Constitutional Reserve Fund, Future Planet Capital and others as well as existing investors, including Oxford Sciences Innovation.	Vaccitech is developing novel immunotherapeutics and vaccines, including TP-300 for chronic hepatitis B infection, which is being tested in a Phase I/II clinical trial that dosed its first patient in February. VTP-200 for persistent, high-risk human papillomavirus infection and VTP-850 in combination with a checkpoint inhibitor for prostate cancer also will move into Phase I/II trials. In addition the company has multiple other preclinical programs in infectious diseases and cancer. Vaccitech co-invented and holds patents to the technology platform used to develop AstraZeneca PLC’s COVID-19 vaccine. (Also see "Biotech Behind Oxford’s Coronavirus Vaccine Welcomes AZ’s Big Pharma 'Muscle'" - Scrip, 1 May, 2020.)
Graphite Bio; South San Francisco	RA Capital Management and Rock Springs Capital led Graphite’s $150m series B round with participation from new investors Cormorant Asset Management, Deerfield Management Company, Federated Hermes Kaufmann Funds, Fidelity Management & Research Company, Janus Henderson Investors, Logos Capital, OrbiMed, Perceptive Advisors, Surveyor Capital (a Citadel company) and Venrock Healthcare Capital Partners as well as existing investor Samsara BioCapital and founding investor Versant Ventures.	The company will use the proceeds to expand its pipeline of targeted gene integration therapies and take its first programs into clinical development, including GPH101 for sickle cell disease, GPH201 for severe combined immune deficiency with IL2RG deficiency (XSCID) and GPH301 for Gaucher disease types 1 and 3. Graphite has investigation new drug (IND) application approval from the US Food and Drug Administration for a Phase I/II clinical trial of GPH101 and is completing IND-enabling studies for GPH201 and GPH103.
Ventyx Biosciences, Inc.; Encinitas, CA	Ventyx said on 9 March that it raised a $114m equity financing led by venBio Partners with participation from new investors, including Third Point, RTW Investments LP, Janus Henderson Investors, Wellington Management, OrbiMed, Surveyor Capital (a Citadel company), Farallon Capital, Vivo Capital, Logos Capital, Qiming Venture PartnersUSA and Cormorant Asset Management plus founding investor New Science Ventures.	The company is developing immune modulators to treat inflammatory disease and autoimmune disorders. It will use the new funding to advance its clinical programs and expand its pipeline of small molecules. Initial program include a S1P1R modulator for inflammatory bowel disease (IBD) and other indications, which was sourced from Oppilan Pharm; an internally developed selective allosteric TYK2 inhibitor for a range of immunological disorders; and an NLRP3 inhibitor program from Zomagen with an initial candidate expected to enter the clinic during the second half of this year.
Neurelis, Inc.; San Diego	The commercial-stage company said on 10 March that it closed a $114m series D round. Investors included new backers Cormorant Asset Management and Decheng Capital along with prior investors LYZZ Capital and HBM Healthcare Investments. Philos & Partners SA organized additional funders.	Neurelis said it will use its fresh capital to fund ongoing commercialization of Valtoco (diazepam nasal spray) for acute treatment of epileptic seizures. The company also will expand its neuroscience pipeline, which includes NRL-2 to control acute panic attacks, NRL-3 to stop seizures that have progressed to status epilepticus, and NRL-4 to address escalation of acute agitation associated with schizophrenia and bipolar 1 mania.
Arctic Vision, Ltd.; Shanghai	Loyal Valley Capital led a series B round that totaled more than $100m, Arctic Vision said on 9 March. Tencent, Octagon Capital and entrepreneur Adrian Cheng from New World Development Group also participated along with existing investors Nan Fung Life Sciences, Pivotal bioVenture Partners China and Morningside Ventures.	Arctic Vision is focused on the development of therapies for pan-ocular disease and will use its new capital to fund drug development, business development, regulatory and commercial activities. The company licensed rights in China and South Korea for a pair of microdrop ophthalmology candidates in the treatment of myopia (ARVN002) and presbyopia (ARVN003) from Eyenovia, Inc. in August. (Also see "Deal Watch: RedHill Finds New Partner For Movantik" - Scrip, 14 Aug, 2020.) Asian rights to ARVN001, Arctic Vision’s candidate for uveitic macular edema, was licensed from Clearside Biomedical, Inc. a year ago; Phase III trials in China are slated to begin this year. (Also see "Deal Watch: Lilly, AbCellera Partner To Attempt Rapid COVID-19 Antibody Development" - Scrip, 13 Mar, 2020.)
Monte Rosa Therapeutics; Boston	The company said on 12 March that it closed a $95m series C roundled by Avoro Capital Advisors with participation from additional new investors Fidelity Management & Research Company LLC, funds and accounts managed by BlackRock, funds and accounts advised by T. Rowe Price Associates Inc., and RTW Investments LP. Founding investor Versant Ventures also participated with other existing investors New Enterprise Associates, Aisling Capital, Cormorant Asset Management, HBM Healthcare Investments, GV, Amzak Health, Sixty Degree Capital, Casdin Capital and Cambridge Asset Management.	Monte Rosa is developing small molecule protein degraders and will use its new financing to take its lead program into the clinic. It also will continue to invest in its technology platform that combines in-house proteomics, structural biology, machine-learning based target selection and computational chemistry capabilities. (Also see "New Treatment Modalities: Protein Degraders And Molecular Glues Gain Traction" - In Vivo, 7 Dec, 2020.) The company has rapidly expanded its capital base, coming out of stealth mode in May of last year with $32.5m in series A cash. (Also see "Finance Watch: VC Mega-Rounds For Machine Learning, CNS Disorders, Academic Discoveries" - Scrip, 1 Jun, 2020.) It closed a $96m series B round four months later. (Also see "Monte Rosa Hits The Heights With $96m Cash Hike" - Scrip, 25 Sep, 2020.)
StrideBio, Inc.; Research Triangle Park, NC	StrideBio announced on 16 March that it closed an $81.5m series B round co-led by Northpond Ventures and Novo Holdings A/S and joined by new investors Pontifax, Octagon Capital, Sarepta Therapeutics, Inc., CaaS Capital and UF Innovate Ventures, along with existing investors Hatteras Venture Partners, UCB Ventures, Takeda Ventures and Alexandria Venture Investments. The company closed a $15.7m series A in 2018. (Also see "Finance Watch: Dementia Discovery Fund Exceeds Fundraising Goal By $150m" - Scrip, 25 Jun, 2018.)	The developer of next-generation adeno-associated virus (AAV)-based gene therapies will use the series B proceeds to take its first programs into the clinic, including four wholly owned candidates for the treatment of monogenic central nervous system (CNS) and cardiovascular diseases. StrideBio also will expand its manufacturing and technological capabilities. The company has established multiple partnerships, including a 2019 deal with Sarepta worth $48m up front for four initial CNS programs. (Also see "Deal Watch: Molecular Templates Aligns With Vertex On Optimizing Stem Cell Candidates" - Scrip, 22 Nov, 2019.)
Exscientia Ltd.; Oxford, UK	Exscientia announced its $60m series C financing in May of last year but revealed on 4 March that the round has grown to $100m with investment from BlackRock, which joins existing series C investors Novo Holdings, Evotec, Bristol Myers Squibb and GT Healthcare Capital. (Also see "Finance Watch: VC Mega-Rounds For Machine Learning, CNS Disorders, Academic Discoveries" - Scrip, 1 Jun, 2020.)	The company is using artificial intelligence to design new drugs and will use the additional series C proceeds to expand its AI-based drug discovery capabilities and expand its drug development pipeline, including taking programs into preclinical and clinical testing. Exscientia counts BMS, Sanofi, Bayer AG, Sumitomo Dainippon Pharma Co., Ltd. and several biotech companies among its collaborators. The company said its headcount more than doubled during the past year and will double again in 2021. (Also see "Two Years Of Artificial Intelligence Deals: A Pharma Snapshot" - Scrip, 30 May, 2019.)
Touchlight; Hampton, UK	The company said on 11 March that it raised £42m ($58m) in a funding round led by Bridford Investments Limited. Touchlight said its financing to date has come from a network of ultra-high net worth individuals, family offices and specialist health care investors.	Touchlight is focused on enzymatic DNA production to enable genetic medicines and will use its new funding to more than triple its manufacturing footprint so that it can help meet global DNA demand, including mRNA and viral vectors for use in COVID-19 vaccines. The company’s capacity expansion, when fully operational in the first quarter of 2022, will be enough to enable more than 1 billion vaccine doses.
OnKure, Inc.; Boulder, CO	OnKure, which closed a $7m series A round in 2018, said on 4 March that it raised $55m in series B financing. Acorn Bioventures with participation from additional new investors Cormorant Asset Management, Surveyor Capital (a Citadel company), Samsara BioCapital, Perceptive Advisors and funds and accounts managed by BlackRock. (Also see "Finance Watch: Lots Of Money, Big And Small, Flowing Into Drug Development" - Scrip, 10 May, 2018.)	The company is developing targeted cancer therapies and will use its series B cash to take its next-generation HDAC inhibitors, including Phase II-ready lead drug candidate OKI-179, into clinical testing. OnKure’s new financing also will fund its pipeline of earlier-stage preclinical molecules focused on epigenetic regulation, delaying resistance and synthetic lethality.
Amphista Therapeutics; Glasgow, UK	The Scottish firm said on 17 March that it closed a $53m (£38m) series B round co-led by Forbion and Gilde Healthcare, and joined by Novartis Venture Fund and Eli Lilly and Company, existing investor BioMotivand founding investor Advent Life Sciences. Amphista closed a $7.5m series A round last year. (Also see "Finance Watch: Biopharma Fundraising Impact Expected, But Not Apparent Yet" - Scrip, 17 Apr, 2020.)	Amphista specializes in targeted protein degradation (TPD) approaches and will use its new funding to accelerate the development of its potent and selective bifunctional molecules known as Amphistas into clinical trials and expand its TPD technology platform. The company is working in a range of diseases, including cancer and CNS diseases.
ExeVir Bio; Ghent, Belgium	The company said on 16 March that it closed a $50m (€42m) series A round led by Fund+ with participation from VIB, UCB Ventures, SFPI-FPIM, V-Bio Ventures and new investors SRIW, Noshaq, Vives IUF, SambrInvest and several Belgian family offices.	ExeVir launched last year to develop llama-derived single domain antibody therapies to protect against viral infections, including XVR011 for the treatment and prevention of COVID-19, which will move into a Phase I/II trial soon. (Also see "ExeVir Bio Looks To Llamas To Tackle Coronavirus Crisis" - Scrip, 28 Jul, 2020.)
Acepodia, Inc.; San Mateo, CA	Acepodia completed a $47m series B round, the company said on 16 March, which was funded by new US institutional investors, including Ridgeback Capital Investments, 8VC and DEFTA Partners as well as Taiwan-based institutional investor CDIB Capital Healthcare. The firm’s series A investors also participated.	The developer of next-generation cancer cell therapies will use its funding to advance off-the-shelf natural killer (NK) cell therapy candidate ACE1702 through clinical development in HER2-expressing solid tumors and to take its preclinical NK and gamma delta T-cell therapy candidates into the clinic. Acepodia out-licensed rights in China, Hong Kong and Macau for ACE1702 and ACE1655 to JW Therapeutics Co., Ltd in July. (Also see "Asia Deal Watch: Promethera Calls On Hao Tian For Asian JV To Advance Its Liver Candidates" - Scrip, 24 Jul, 2020.)
Bicara Therapeutics ; Cambridge, MA	Bicara launched on 15 March with $40m in seed financing from the Indian pharmaceutical firm Biocon, Ltd.	The start-up is developing dual-action biologics that are designed to spur potent and durable immune responses in the tumor microenvironment. Lead drug candidate BCA101 is an EGFR/TGFbeta-trap bifunctional antibody that is being tested in a Phase I/II solid tumor study as monotherapy and in combination with Merck & Co., Inc.’s PD-1 inhibitor Keytruda (pembrolizumab). Dose expansion in the study is expected to begin in the second half of 2021.
Eirion Therapeutics; Woburn, MA	The aesthetic dermatology company said on 9 March that it raised $40m in financing from Shanghai Haohai Biological Technology Ltd., including a $32m series A preferred stock investment ($31m from Haohai and $1m from a US investor) in Eirion and an $8m upfront payment for rights in China to all of the US company’s products. Eirion previously was funded with $11m in convertible notes that now will convert into series A preferred stock.	Eirion could earn approximately$43m in development fees and $444m in sales milestone payments, plus royalties from the high single digits to low twenties under its deal in China with Haohai, which pertains to Eirion's topical neuromodulator ET-01 and injectable botulinum product candidate AI-09 for aesthetic indications. Haohai also has Chinese rights to the small molecules ET-02 (topical) and ET-03 (oral) for hair loss and hair greying. Eirion was spun out of Anterios, Inc. when Allergan plc acquired Anterios in 2016. (Also see "Allergan Bolsters Blockbuster Botox With Topical Toxin Buy" - Scrip, 8 Jan, 2016.)
Visus Therapeutics, Inc.; Seattle	Visus said on 9 March that it raised $36m in series A financing on from Johnson & Johnson Innovation-JJDC Inc., RTW Investments LP and Wille AG with participation from the company’s existing shareholders.	The company is trying to develop the first presbyopia-correcting eyedrop that has the potential to last for a minimum of eight hours. Lead drug candidate Brimochol, a proprietary combination of carbachol and brimonidine tartrate, is designed to be a once-daily eye drop to correct loss of near vision due to presbyopia. The drug already has been tested in more than 200 patients and Visus will file an IND application with the US FDA during the first quarter of 2021 to facilitate additional trials under the agency’s 505(b)2 pathway for therapies based on previously approved drugs.
Affinivax, Inc.; Cambridge, MA	CARB-X, a global non-profit working to accelerate R&D for antibacterials against drug-resistant bacteria, granted an award of up to $22m to Affinivax to fund development of a vaccine against Staphylococcus aureus bacterial infections. The company closed a $226m series C round in January to fund development of vaccines with its Multiple Antigen Presenting System (MAPS) platform. (Also see "Finance Watch: Scorpion Closes $162m Series B, Bringing Recent Total To $270m" - Scrip, 8 Jan, 2021.)	The CARB-X funding will fund Affinivax’s advancement of an S. aureus vaccine through an initial clinical trial. The money will be doled out to the company based on the achievement of preclinical, manufacturing and clinical milestones. A lead MAPS vaccine candidate against S. aureus was developed and tested preclinically at Boston Children’s Hospital. Affinivax is moving the program towards an IND filing with the US FDA.
CalciMedica, Inc.; San Diego	CalciMedica revealed on 11 March that it closed a $21m series D round led by Quark Venture LP and GHS Fund (Quark Venture LP and GF Securities) and joined by previous investors, Valence Life Sciences and Sanderling Ventures.	The company is developing drugs thattarget calcium release-activated calcium (CRAC) channels for the treatment of severe acute and chronic inflammatory diseases. Proceeds from CalciMedica’s financing will support clinical development of its CRAC channel inhibitors including Auxora, which is being evaluated in a placebo-controlled clinical trial in severe and critical COVID-19 pneumonia and in a Phase IIb trial that soon will begin enrolling patients with acute pancreatitis.
NeoPhore Limited; Cambridge, UK	NeoPhore, spun out of PhoreMost Ltd. in 2017 with initial funding from Sixth Element Capital LLP, said on 9 March that it closed a £15.2m ($21m) series B round led by Claris Ventures. (Also see "NeoPhore Enters Immuno-Oncology Fray With DNA-Repair Technology" - Scrip, 8 Feb, 2018.) Founding investor CRT Pioneer Fund also participated in the series B along with 2Invest, 3B Future Health Fund and Astellas Venture Management.	The company is developing small molecules that target the DNA mismatch repair (MMR) pathway to treat cancer. NeoPhore will use its financing to advance its drug discovery pipeline based on the concept that inhibition of MMR in tumors generates neoantigens that the patient’s immune system can target with a powerful immune response.
Delonix Bioworks; Shanghai	The Chinese firm revealed on 11 March that it raised $14m in seed financing from lead investors Boehringer Ingelheim Venture Fund and IDG Capital with participation from ZhenFund and an undisclosed investor.	Delonix said that it will use the funding to accelerate its efforts to build synthetic biology vaccine platforms and advance vaccine candidates into clinical trials. The company’s synthetic biology and antigen discovery platforms enable rational design and programming of bacteria into safer and more effective vaccines with an initial program focused on antimicrobial resistance.
Immune Biosolutions; Sherbrooke, Quebec, Canada	Immune Biosolutions said on 16 March that it received C$13.44m ($10.8m) from Canada’s Strategic Innovation Fund.	The funding will help the company take its immunotherapy – an antibody with the potential to neutralize and eliminate COVID-19 and its variants – into Phase II clinical trials and build manufacturing capabilities for its antibody programs.