Acuitas Therapeutics

Canadian biotechnology company

Acuitas Therapeutics Inc.

Formerly| AlCana Technologies
Company type| Private
Industry|

* Biotechnology
* Pharmaceutical

Founded| February 2009; 16 years ago (2009-02)
Founder|

* Thomas Madden
* Pieter Cullis
* Michael Hope

Headquarters| Vancouver, Canada
Website| acuitastx.com

Acuitas Therapeutics Inc. is a Canadian biotechnology company based in Vancouver, British Columbia. The company was established in February 2009 to specialize in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticle (LNP) technology, a key component of the mRNA vaccines deployed for COVID-19.[1][2]

## History

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### Inex Pharmaceuticals

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Pieter Cullis was among several scientists experimenting with liposomes in the early 1980s, fascinated by their properties in many aspects of human biology.[3] Cullis discovered he could load cancer drugs into liposomes, inspiring him and several colleagues at the University of British Columbia to form Inex Pharmaceuticals in 1992.[4] One of Inex's co-founders was Thomas Madden, who had recently emigrated to Canada from the United Kingdom to complete post-doctoral studies in biochemistry.[5]

### Tekmira Pharmaceuticals

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To improve the prospects of successfully advancing gene therapy techniques, Cullis developed and patented a new form of lipid nanoparticle designed to deliver genetic material to recipient cells.[6] From 1994 onward, a series of patents were filed describing the technology Cullis and his colleagues created, including the addition of a chemical called polyethylene glycol (PEG) to further increase the LNP's ability to pass into cells.[7][8][9][10][11][12]

Several spinoff companies emerged based on the research and intellectual property, including Protiva Biotherapeutics (which focused on gene therapies based on RNAi).[13] In 2001, Inex was developing topotecan with GlaxoSmithKline and by 2004 was in the research stage of preparing their own targeted cancer vaccine program.[14][15] However, Cullis left the company after the Food and Drug Administration declined to approve their chemotherapy products.[16] Inex then downsized and rebranded to Tekmira Pharmaceuticals.[17] This led to $28.5 million in funding from “large biotechnology companies” for further development of anti-cancer drugs and RNA therapeutics for other diseases.[18]

### AlCana and Acuitas

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Madden, Cullis and Michael Hope then founded AlCana Technologies in February 2009, later changing its name to Acuitas Therapeutics.[2] With early financial support from the National Research Council Canada's Industrial Research Assistance Program (NRC IRAP), the company spent several years establishing its research and development capabilities and building out its staff.[1] This led to its development of a viable lipid nanoparticle platform that showed promise for use in a variety of medical applications including chemotherapy, gene therapy and genetic vaccines. By 2012, the company had decided to focus on LNP development for the delivery of mRNA.[19]

The Natural Research Council Canada awarded Acuitas a $173,020 grant in September 2013 and a $498,640 grant in February 2015 to continue their research and development.[20][21]

In April 2016, Acuitas entered into a development and option agreement with German biotechnology company CureVac to jointly develop a product incorporating Acuitas' LNP technology with CureVac's mRNA materials.[22][23] The encapsulated mRNA product described under the agreement was commissioned for use in in vitro and animal testing for pre-clinical trials of a vaccine candidate. Acuitas also worked on developing an mRNA vaccine against rabies.[24]

### COVID-19

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According to president and CEO Thomas Madden, Acuitas began working towards a COVID-19 vaccine in February 2020. The company also partnered with CureVac and Imperial College London for their own vaccine trials.[25][24] Madden noted that the mRNA vaccine platform would allow for rapid development of updated vaccines in case the SARS-CoV-2 virus evolved beyond the immunity generated by the initial formulation, though additional clinical testing would still be required.[24] He also stated that the Pfizer–BioNTech COVID-19 vaccine would stop the spread of the virus and infection in vaccinated individuals.

On December 9, 2020, Acuitas released a statement celebrating Health Canada's approval of Pfizer and BioNTech's BNT162b2 COVID-19 vaccine,[26] stating they had “made history” due to their collaboration through the LNP delivery system, “a key element… in the development of this vaccine.”[27] As a privately held company, Acuitas doesn't publicly publish its earnings. However, Madden stated the company's annual revenues were "more than tens of millions of dollars" as of the end of 2020, having increased by 75% in the prior several years leading up to the COVID-19 crisis and expectations to grow by "at least that amount" through to 2022.[5]

### Gene-editing therapies

[edit]

Acuitas entered a long-term partnership with Science World in January 2022.[28][29] In April 2022, Acuitas completed an agreement with Korean biotechnology company GC Biopharma to develop vaccines and therapeutics using their joint LNP-mRNA platform.[30] In June 2022, Acuitas donated $25,000 to the Brain Tumour Foundation of Canada to create the Frazer Anderson Pediatric Research Grant.[31] The company entered an agreement with genetic editing company Arbor Biotechnologies in August 2022 to target rare liver diseases, combining Acuitas' LNP technology with Arbor's CRISPR gene editing capabilities.[32]

In June 2023, Bayer announced it had entered a partnership with Acuitas to use its lipid nanoparticles to deliver in vivo gene editing products to the liver.[33]

On May 15, 2025, multiple sources reported that Acuitas lipid nanoparticles had been used to deliver a personalized CRISPR gene-editing therapy designed to treat a baby with a rare genetic disease, representing the first time CRISPR technology has been successfully used as a "bespoke" therapeutic for a single patient.[34][35] The FDA-approved therapy, which was developed by a team of researchers at the Children's Hospital of Philadelphia, University of Pennsylvania, and the Innovative Genomics Institute,[36][37] and included RNA manufactured by Danaher companies Aldevron and Integrated DNA Technologies (IDT), was customized to the child's individual DNA; specifically it aimed to address a gene mutation causing severe carbamoyl phosphate synthetase 1 (CPS1) deficiency.[38]

## Organization

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### Partners

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Acuitas has partnered with several pharmaceutical companies and universities to collaboratively advance specific product candidates, as well as to support the broader development of the personalized medicine sector. It is a member of BIOTECanada, a biotechnology industry association,[39] and a sponsor of Life Sciences British Columbia.[40] It has participated in research published by the University of Pennsylvania, Mount Sinai Health System, Chulalongkorn University, Thomas Jefferson University, and BioNTech.[41]

Acuitas is a sponsor of the Vancouver-based Student Biotechnology Network.[42]

### Ownership

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Rumours circulated in early 2022 that Prime Minister Justin Trudeau owned a 40% stake in Acuitas, either directly or through the Trudeau Foundation.[43] This was refuted by the Office of the Prime Minister, CEO Thomas Madden and co-founder Pieter Cullis.[44]

## References

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1. ^ a b "Producing lipid nanoparticle technology for vaccine delivery (Acuitas Therapeutics)". National Research Council Canada. 2021-05-17. Archived from the original on 2022-03-02. Retrieved 2022-09-30.
2. ^ a b "Our History". Acuitas Therapeutics. Archived from the original on 2022-03-07. Retrieved 2022-09-30.
3. ^ Horejs, Christine (2021-09-21). "From lipids to lipid nanoparticles to mRNA vaccines". Nature Reviews Materials. 6 (12): 1075–1076\. Bibcode:2021NatRM...6.1075H. doi:10.1038/s41578-021-00379-9. ISSN 2058-8437. PMC 8454296. PMID 34567796.
4. ^ "Welcome to Inex Pharmaceuticals Corporation". Inex Pharmaceutical Corporation. Archived from the original on 2000-06-04. Retrieved 2022-10-03.
5. ^ a b Silcoff, Sean (2020-12-02). "Inside Acuitas Therapeutics, the Vancouver company providing a crucial piece of Pfizer's COVID-19 vaccine". The Globe and Mail. Retrieved 2022-10-03.
6. ^ Kulkarni, Jayesh A.; Cullis, Pieter R.; van der Meel, Roy (2018-06-01). "Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility". Nucleic Acid Therapeutics. 28 (3): 146–157\. doi:10.1089/nat.2018.0721. ISSN 2159-3337. PMID 29683383.
7. ^ Choi, Lewis Siu Leung; Madden, Thomas D.; Webb, Murray S. (1994-09-30). "Polyethylene glycol modified ceramide lipids and liposome uses thereof". Canada Patent Database. Archived from the original on 2022-03-08. Retrieved 2022-10-03.
8. ^ Holland, John W.; Cullis, Pieter R.; Madden, Thomas D. (1994). "Bilayer stabilizing components and their use in forming programmable fusogenic liposomes (Canadian Intellectual Property Office Patent No. 2201121)". Canadian Patent Database. Archived from the original on 2022-03-08. Retrieved 2022-10-03.
9. ^ Cullis, Pieter R.; Fenske, David B.; Hope, Michael J.; Wong, Kim F. (1995-02-27). "Method for loading lipid vesicles (Canadian Intellectual Property Office Patent No. CA 2213861)". Canadian Patent Database. Archived from the original on 2022-03-08. Retrieved 2022-10-03.
10. ^ Cullis, Pieter R.; Choi, Lewis S.L.; Monck, Myrna; Bailey, Austin (1996-04-11). "Fusogenic liposomes (Canadian Intellectual Property Office Patent No. CA 2252055)". Canadian Patent Database. Archived from the original on 2022-03-08. Retrieved 2022-10-03.
11. ^ Cullis, Pieter R.; Fenske, David B.; Maclachlan, Ian (2000-10-25). "Lipid formulations for target delivery (Canadian Intellectual Property Office Patent No. CA 2426244)". Canadian Patent Database. Archived from the original on 2022-03-08. Retrieved 2022-10-03.
12. ^ "Patents Assigned to Acuitas Therapeutics Inc. - Justia Patents Search". Justia. Archived from the original on 2022-10-21. Retrieved 2022-10-21.
13. ^ Vardi, Nathan (2021-08-17). "Covid's Forgotten Hero: The Untold Story Of The Scientist Whose Breakthrough Made The Vaccines Possible". Forbes. Archived from the original on 2022-03-22. Retrieved 2022-10-03.
14. ^ Garcia, Joseph A. (2001-12-13). "Inex Pharmaceuticals Completes Bought Deal of Common Shares ($43 million)". Lexpert. Archived from the original on 2022-03-22. Retrieved 2022-10-03.
15. ^ Main, David J. (2005-03-17). "2004 Annual Report". Morningstar, Inc. Inex Pharmaceuticals Corporation. Archived from the original on 2022-03-22. Retrieved 2022-10-03.
16. ^ "Inex Pharmaceuticals slashes more staff as CEO departs". CBC News. 2005-06-21. Archived from the original on 2022-03-22. Retrieved 2022-10-03.
17. ^ "INEX Pharmaceuticals Provides Timeline for Completing Spin-out of Tekmira Pharmaceuticals Corporation". BioSpace. 2007-02-26. Archived from the original on 2022-03-22. Retrieved 2022-10-03.
18. ^ Mortimer, Ian; Rizgalla, Nicole; Reid, Sheldon (2007-05-01). "Inex Pharmaceuticals announces completion of spin-out of Tekmira Pharmaceuticals Corporation". Arbutus Biopharma. Archived from the original on 2022-10-03. Retrieved 2022-10-03.
19. ^ "Acuitas Therapeutics Inc. v. Genevant Sciences GMBH, and Arbutus Biopharma Corp" (PDF). Reuters. United States District Court for the Southern District of New York. 2022-03-18. Archived (PDF) from the original on 2024-01-02.
20. ^ Treasury Board of Canada Secretariat. "GC-2013-Q3-23969". Government of Canada. Archived from the original on 2022-03-08. Retrieved 2022-10-01.
21. ^ Treasury Board of Canada Secretariat. "GC-2014-Q4-26252". Government of Canada. Archived from the original on 2022-03-08. Retrieved 2022-10-01.
22. ^ "Development and Option Agreement between CureVac AG and Acuitas Therapeutics Inc". Securities and Exchange Commission. 2016-04-29. Archived from the original on 2022-09-30. Retrieved 2022-09-30.
23. ^ Plieth, Jacob; Elmhirst, Edwin (2020-07-27). "Curevac muddies the Moderna/Arbutus waters further". Evaluate Vantage. Archived from the original on 2021-10-10. Retrieved 2022-09-30.
24. ^ a b c Bianchini, Elisabetta (2020-11-10). "Acuitas Therapeutics: The Canadian technology that the Pfizer and BioNTech COVID-19 vaccine 'can't work without'". Yahoo! News. Archived from the original on 2022-09-30. Retrieved 2022-09-30.
25. ^ Lowe, Derek (2021-06-17). "CureVac Comes Up Short". Science. Archived from the original on 2022-09-30. Retrieved 2022-09-30.
26. ^ Health Canada (9 December 2020). "Comirnaty (tozinameran)". COVID-19 vaccines and treatments portal. Archived from the original on 2022-03-07. Retrieved 2022-09-30.
27. ^ "Vancouver-based Acuitas Therapeutics Makes History with First COVID-19 Vaccine Approved for Use in Canada" (PDF). Acuitas Therapeutics. 2020-12-09. Archived (PDF) from the original on 2022-04-28. Retrieved 2022-09-30.
28. ^ Acuitas Therapeutics (2022-01-24). "Acuitas Announces Key Partnership with Science World". Life Sciences British Columbia. Archived from the original on 2022-09-29. Retrieved 2022-09-29.
29. ^ Slusarchuk, Cheryl (2022-05-12). "2021/22 Annual Report" (PDF). Science World. Archived (PDF) from the original on 2022-09-28. Retrieved 2022-09-29.
30. ^ Han-soo, Lee (2022-04-29). "GC to license in lipid nanoparticle delivery system from Canadian firm". KBR. Archived from the original on 2022-10-02. Retrieved 2022-10-02.
31. ^ Allison, Greg (2022-09-29). "Fight Like Frazer". Brain Tumour Foundation of Canada. Archived from the original on 2023-07-07. Retrieved 2022-10-02.
32. ^ "Arbor Partners with Acuitas for Lipid Nanoparticle Delivery System for Use in Rare Liver Diseases". Global Genes. 2022-08-25. Archived from the original on 2022-08-31. Retrieved 2022-10-02.
33. ^ Taylor, Nick Paul (2023-06-07). "Bayer bags access to gene editing delivery tech from biotech behind Pfizer's COVID-19 vaccine". Fierce Biotech. Archived from the original on 2023-07-08. Retrieved 2023-07-08.
34. ^ Park, Alice (2025-05-15). "A Baby Receives the First Customized CRISPR Treatment". TIME. Retrieved 2025-05-16.
35. ^ Ledford, Heidi (2025-05-15). "World's first personalized CRISPR therapy given to baby with genetic disease". Nature. doi:10.1038/d41586-025-01496-z. ISSN 1476-4687. PMID 40374912.
36. ^ "Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment". National Institutes of Health (NIH). 2025-05-15. Retrieved 2025-05-16.
37. ^ Murdock, Andy (2025-05-15). "First Patient Treated with Personalized CRISPR Therapy, Developed in Just Six Months". Innovative Genomics Institute (IGI). Retrieved 2025-05-16.
38. ^ Musunuru, Kiran; Grandinette, Sarah A.; Wang, Xiao; Hudson, Taylor R.; Briseno, Kevin; Berry, Anne Marie; Hacker, Julia L.; Hsu, Alvin; Silverstein, Rachel A.; Hille, Logan T.; Ogul, Aysel N.; Robinson-Garvin, Nancy A.; Small, Juliana C.; McCague, Sarah; Burke, Samantha M. (2025). "Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease". New England Journal of Medicine. 392 (22): 2235–2243\. doi:10.1056/NEJMoa2504747. ISSN 0028-4793. PMID 40373211.
39. ^ "Member Listings". BIOTECanada. 2021-12-23. Archived from the original on 2022-09-01. Retrieved 2022-10-01.
40. ^ "Our Sponsors". Life Sciences British Columbia. Archived from the original on 2022-04-25. Retrieved 2022-10-01.
41. ^ "Acuitas Therapeutics". Nature Index. 2022-06-30. Archived from the original on 2022-10-02. Retrieved 2022-10-02.
42. ^ "2020/21 Sponsors". Student Biotechnology Network. Archived from the original on 2022-10-02. Retrieved 2022-10-02.
43. ^ Sadeghi, McKenzie (2022-02-24). "Fact check: Viral rumor falsely claims the Trudeau Foundation owns 40% of Acuitas Therapeutics". USA TODAY. Archived from the original on 2022-09-05. Retrieved 2022-10-02.
44. ^ "Fact Check-Neither Justin Trudeau nor the Trudeau Foundation have shares in Acuitas Therapeutics". Reuters. 2022-02-18. Archived from the original on 2023-07-07. Retrieved 2022-10-02.

## External links

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* Official website