Company:Precision BioSciences

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Short description: American gene editing company
Precision BioSciences, Inc.
Industry
Founded2006; 18 years ago (2006) in Durham, North Carolina
Founders
  • Derek Jantz
  • Jeff Smith
  • Matt Kane
Key people
Michael Amoroso (President and CEO)
Revenue$115.5 million[1]
Number of employees
192[2] (2022)
Websiteprecisionbiosciences.com

Precision BioSciences, Inc. is a publicly traded American clinical stage gene editing company headquartered in Durham, North Carolina.[3] Founded in 2006, Precision is focused on developing both in vivo and ex vivo gene editing therapies using its proprietary "ARCUS" genome editing platform.[4]

History

Derek Jantz and Jeff Smith met as postdoctoral fellows at Duke University,[5] and in March 2006, they founded Precision BioSciences along with Matt Kane, a student at the Duke Fuqua School of Business at the time.[3] The company went through two rounds of early funding: a Series A round led by venBio to fund development of the genome editing platform,[6] and Series B financing to fund product development efforts.[7][8] The company completed its initial public offering in 2019, and trades under the Nasdaq ticker DTIL.[9][10]

Precision entered into a partnership with Eli Lilly in November 2020 to use ARCUS editing for up to six in vivo targets connected to genetic disorders,[11] beginning with Duchenne muscular dystrophy.[12] In September 2021, Precision announced two more collaborations, with UK biotechnology company Tiziana Life Sciences to explore using foralumab to aid chimeric antigen receptor (CAR) T cell therapy,[13] and with Philadelphia-based iECURE to advance candidates into clinical trials and investigate how ARCUS can help treat liver diseases.[11] Michael Amoroso, the former CEO of cell and gene therapy developer Abeona Therapeutics, succeeded Matt Kane as President and CEO in October 2021.[3] That December, Precision announced its entry into an agreement with a syndicate of investors led by ACCELR8 to spin off its subsidiary, Elo Life Systems, and create an independent company focused on food and agriculture business.[14]

ARCUS genome editing

Precision BioSciences' proprietary technology is the ARCUS platform and ARCUS nucleases.[4][8] ARCUS nucleases are based on a naturally occurring genome editing enzyme, I-CreI, a homing endonuclease that evolved in the algae Chlamydomonas reinhardtii[4][12] to make highly specific cuts and DNA insertions in cellular DNA.[15] The nuclease is able to deactivate itself once gene edits are made, which minimizes potential off-targeting.[12][16] An ARCUS nuclease is also much smaller in size than CRISPR spCas9.[17] It can use either adeno-associated virus (AAV) vectors or lipid nanoparticles (LNPs) for delivery to specific tissues and cells.[18] Precision has used ARCUS nucleases to develop multiple ex vivo allogeneic, "off-the-shelf" CAR T cell immunotherapies in early-stage clinical trials.[4][19] The company also uses ARCUS for in vivo gene editing programs,[4] some of which are in preclinical development as of May 2022.[20][18]

Similar to I-CreI, ARCUS nucleases generate a unique cleavage site in DNA that is characterized by four-base-pair, 3' overhangs.[4] ARCUS nucleases can perform a range of complex edits, including gene insertion, gene excision, and gene repair.[8][15] ARCUS nucleases are able to enact all editing operations in one step, which enables efficient multiplexing of edits.[19]

Precision has demonstrated some additional applications of the ARCUS platform, including treating ornithine transcarbamylase deficiency in newborn nonhuman primates and in the use of a LNP to treat chronic Hepatitis B.[15] The company is also pursuing PBGENE-PCSK9, a candidate to treat familial hypercholesterolemia, and PBGENE-PH1, a candidate to treat primary hyperoxaluria type 1.[21][22]

Clinical trials

Precision is in the process of developing multiple candidates targeting non-Hodgkin lymphoma, acute lymphoblastic leukemia (ALL),[4][19] and multiple myeloma.[23] The company's lead candidate targeting CD19, PBCAR0191,[19] received orphan drug designation from the U.S. Food and Drug Administration for the treatment of ALL and mantle cell lymphoma, an aggressive subtype of non-Hodgkin lymphoma, as well as fast track designation for the treatment of B-cell ALL.[23] PBCAR0191 began its Phase 1/2a clinical trial of adult subjects in March 2019.[24][23] In June 2022, Precision reported a 100% response rate, a 73% complete response rate, and a 50% durable response rate, and the company sought to increase enrollment in the study.[25][26]

Precision is also developing PBCAR19B as an anti-CD19[26] stealth cell candidate that employs a single gene edit to knock down beta-2 microglobulin, for which a Phase 1 study began in June 2021.[27][28] The company is also conducting a Phase 1/2a clinical trial evaluating PBCAR269A, its investigational allogeneic B-cell maturation antigen-targeted CAR T cell therapy, for the treatment of multiple myleloma.[26] PBCAR269A began its Phase 1 trials in April 2020,[29] and as of July 2022 had moved onto recruitment for its Phase 1/2a study, which features PBCAR269A in combination with nirogacestat, a gamma secretase inhibitor.[26] In 2020, the FDA granted fast track designation to PBCAR269A for the treatment of relapsed or refractory multiple myeloma, having previously provided orphan drug designation.[23]

References

  1. ↑ "Precision BioSciences Reports Fourth Quarter and Fiscal Year 2021 Financial Results and Provides Business Update". Precision BioSciences (Press release). 15 March 2022. Retrieved 9 August 2022.
  2. ↑ "Form 10-Q: Quarterly report which provides a continuing view of a company's financial position". Precision BioSciences. 30 June 2022. https://investor.precisionbiosciences.com/node/9451/html. 
  3. ↑ 3.0 3.1 3.2 Ezzone, Zac (24 February 2022). "Executive voice: New leader joins Durham biotech chasing permanent cures". Triangle Business Journal. https://www.bizjournals.com/triangle/news/2022/02/24/executive-voice-michael-amoroso-precision-bio.html. 
  4. ↑ 4.0 4.1 4.2 4.3 4.4 4.5 4.6 Shaffer, Catherine (5 January 2022). "Novel Gene Editing Systems Come Into Their Own". Genetic Engineering and Biotechnology News. https://www.genengnews.com/topics/genome-editing/novel-gene-editing-systems-come-into-their-own/. 
  5. ↑ Graff, Gregory D.; Sherkow, Jacob S. (2020-08-31). "Models of Technology Transfer for Genome-Editing Technologies" (in en). Annual Review of Genomics and Human Genetics 21 (1): 509–534. doi:10.1146/annurev-genom-121119-100145. ISSN 1527-8204. PMID 32151165. https://www.annualreviews.org/doi/10.1146/annurev-genom-121119-100145. 
  6. ↑ Saxena, Varun (12 May 2015). "Gene editing player gets $25M in Series A round" (in en). Fierce Pharma. https://www.fiercepharma.com/drug-delivery/gene-editing-player-gets-25m-series-a-round. 
  7. ↑ Terry, Mark (26 June 2018). "Precision BioSciences Closed on a $110 Million Series B Financing" (in en-US). https://www.biospace.com/article/precision-biosciences-closed-on-a-110-million-series-b-financing/. 
  8. ↑ 8.0 8.1 8.2 Taylor, Nick Paul (26 June 2018). "Precision BioSciences bags $110M to take off-the-shelf CAR-T into humans" (in en). https://www.fiercebiotech.com/precision-biosciences-bags-110m-to-take-off-shelf-car-t-into-humans. 
  9. ↑ "Precision Biosciences S-1 Filing". 1 March 2019. https://www.sec.gov/Archives/edgar/data/1357874/000119312519061192/d717633ds1.htm. 
  10. ↑ Terry, Mark (28 March 2019). "Precision BioSciences Launches IPO to Raise $126.4 Million" (in en-US). https://www.biospace.com/article/precision-biosciences-launches-ipo-to-raise-126-4-million-/. 
  11. ↑ 11.0 11.1 Terry, Mark (9 September 2021). "Precision Signs Another Collab for ARCUS Genome Editing Platform". BioSpace. https://www.biospace.com/article/precision-biosci-partners-with-iecure-and-describes-clinical-development-program/. 
  12. ↑ 12.0 12.1 12.2 Idrus, Amirah Al (20 November 2020). "Lilly, Precision Biosciences team up on Duchenne gene therapy in $135M deal" (in en). Fierce Biotech. https://www.fiercebiotech.com/biotech/lilly-precision-biosciences-team-up-duchenne-gene-therapy-135m-deal. 
  13. ↑ "Precision BioSciences and Tiziana Life Sciences partner on foralumab". The Pharma Letter. 2 September 2021. https://www.thepharmaletter.com/article/precision-biosciences-and-tiziana-life-sciences-partner-on-foralumab. 
  14. ↑ Ezzone, Zac (27 December 2021). "Durham biotech spins out food, agriculture company". Triangle Business Journal. https://www.bizjournals.com/triangle/news/2021/12/27/durham-biotech-spins-out-food-agriculture-company.html. 
  15. ↑ 15.0 15.1 15.2 Bayer, Max (May 23, 2022). "Precision BioSciences makes case for 2 gene editing programs over CRISPR with preclinical data". Fierce Biotech. https://www.fiercebiotech.com/research/precision-biosciences-boasts-preclinical-data-two-gene-editing-programs-making-its-case. 
  16. ↑ Weintraub, Arlene (29 September 2021). "In vivo gene editing grabs the spotlight after Intellia triumph, but challenges loom" (in en). Fierce Biotech. https://www.fiercebiotech.com/research/vivo-gene-editing-grabs-spotlight-after-intellia-triumph-but-challenges-loom. 
  17. ↑ Meng, Da; Ragi, Sara D.; Tsang, Stephen H. (2020). "Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa". Molecular Therapy 28 (10): 2139–2149. doi:10.1016/j.ymthe.2020.08.012. PMID 32882181. ""Given that Cas9 genes themselves are approaching the size limit of AAV vectors, usage of long promoters to increase specificity and expression level is prohibited. ... ARCUS meganuclease is much smaller (~300 aa) and does not require gRNAs, making it easier to deliver via a single AAV vector."". 
  18. ↑ 18.0 18.1 Gorsuch, Cassandra L.; Nemec, Paige; Yu, Mei; Xu, Simin; Han, Dong; Smith, Jeff; Lape, Janel; Van Buuren, Nicholas et al. (2022). "Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo". Molecular Therapy 30 (9): 2909–2922. doi:10.1016/j.ymthe.2022.05.013. PMID 35581938. 
  19. ↑ 19.0 19.1 19.2 19.3 Amorosi, Drew (10 August 2021). "One-step gene-editing technique opens door to safe, effective off-the-shelf CAR T cells" (in en). Healio. https://www.healio.com/news/hematology-oncology/20210810/onestep-geneediting-technique-opens-door-to-safe-effective-offtheshelf-car-t-cells. 
  20. ↑ Sarkar, Anjali A. (23 February 2021). "Preclinical Study Shows Safety, Efficacy, and Durability of Lowering LDL-Cholesterol Levels Long-Term in Non-Human Primates". GEN - Genetic Engineering and Biotechnology News. https://www.genengnews.com/news/preclinical-study-shows-safety-efficacy-and-durability-of-lowering-ldl-cholesterol-levels-long-term-in-non-human-primates/. 
  21. ↑ Haskins, Bryant (18 September 2021). "Precision Biosciences inks gene editing deal with iECURE". WRAL TechWire. https://wraltechwire.com/2021/09/18/precision-biosciences-inks-gene-editing-deal-with-iecure/. 
  22. ↑ "Precision and Iecure ink collaboration deal". The Science Advisory Board. 9 September 2021. https://www.scienceboard.net/index.aspx?sec=sup&sub=Drug&pag=dis&ItemID=3254. 
  23. ↑ 23.0 23.1 23.2 23.3 "CAR T-cell Therapies for the Treatment of Patients with Acute Lymphoblastic Leukemia". Onco'Zine. 13 October 2020. https://www.oncozine.com/car-t-cell-therapies-for-the-treatment-of-patients-with-acute-lymphoblastic-leukemia/. 
  24. ↑ Precision BioSciences, Inc. (2022-04-18). Phase 1/2a, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate Safety and Clinical Activity of PBCAR0191 in Subjects With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) and r/r B-cell Acute Lymphoblastic Leukemia (B-ALL). https://clinicaltrials.gov/ct2/show/NCT03666000. 
  25. ↑ Bayer, Max (June 8, 2022). "Precision hails phase 1/2 data as proof it could deliver the first approved off-the-shelf CAR-T". https://www.fiercebiotech.com/clinical-data/precision-hails-early-data-its-shelf-car-t-lifeline-sickest-patients. 
  26. ↑ 26.0 26.1 26.2 26.3 "Precision BioSciences Releases Data on CAR T Therapy Candidates" (in en). 13 June 2022. https://www.pharmtech.com/view/precision-biosciences-releases-data-on-car-t-therapy-candidates. 
  27. ↑ McConaghie, Andrew (30 March 2022). "'We'll Be First With Off-The-Shelf CAR-T,' Vows Precision Biosciences – And Hopes To Win Over Gene-Editing Skeptics". Informa Pharma Intelligence. https://pink.pharmaintelligence.informa.com/SC146135/Well-Be-First-With-OffTheShelf-CART-Vows-Precision-Biosciences--And-Hopes-To-Win-Over-GeneEditing-Skeptics. 
  28. ↑ Precision BioSciences, Inc. (2022-03-01). A Phase 1 Study of PBCAR19B in Participants With CD19-expressing Malignancies. https://clinicaltrials.gov/ct2/show/NCT04649112. 
  29. ↑ Precision BioSciences, Inc. (2021-06-24). A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion Study to Evaluate the Safety and Clinical Activity of PBCAR269A, With or Without Nirogacestat, in Study Participants With Relapsed/Refractory Multiple Myeloma. https://clinicaltrials.gov/ct2/show/NCT04171843. 

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