ProQR

ProQR Therapeutics NV (NASDAQ: PRQR) is a Dutch biotechnology company based in Leiden, the Netherlands. In addition to its headquarters in Leiden, the company also has a presence in Cambridge, Massachusetts.[1] The company was funded in 2012 by chief executive officer (CEO) Daniel A. de Boer. It specializes in the development of RNA therapeutics using its RNA editing platform technology called Axiomer.[2]

ProQR
TypePublic
Nasdaq: PRQR
IndustryBiotechnology Pharmaceuticals
Founded2012
Headquarters
Leiden
Key people
Daniel de Boer (CEO)
ProductsRNA therapeutics
Number of employees
  • 150
Websitehttp://www.proqr.com/

History

ProQR was founded in 2012 by chief executive officer (CEO) Daniel A. de Boer and co-founders Henri Termeer, Dinko Valerio and Gerard Platenburg.[3][4][5][6]

The initial pipeline of the company was to develop small-molecule drugs or gene therapy that would treat cystic fibrosis (CF).[7] Positive proof of concept (PoC) was achieved in 2016 with the clinical trial of the molecule QR-010 targeting the gene coding for cystic fibrosis transmembrane conductance regulator (CFTCR) in patients.[7] Subsequently, the company expanded its pipeline into treating other rare diseases, including ophthalmology.[7]

In 2017, a spin-out of ProQR named Amylon Therapeutics was established in Leiden with the focus on the development of therapies for the central nervous system.[8]

In 2021, ProQR announced a collaboration with Eli Lilly and Company on the Axiomer technology focused on "genetic disorders in the liver and nervous system". They signed a deal with which ProQR would receive $50 million ($20 million upfront) and an equity investment of $30 million.[9] The relationship between the two firms "deepened" in 2022 with expansion of the indications engagement and a $75 million cash infusion which included an equity stake.[10]

In 2022, the company announced it would focus exclusively on advancing its RNA editing technology.[11]

Projects

ProQR is an RNA editing company targeting genetic diseases by focusing on making changes to the RNA.[12] The firm's RNA editing technology, called Axiomer can make targeted single nucleotide changes to RNA.[13] ProQR’s proprietary Axiomer technology is based on short strands of synthetic RNA called editing oligonucleotides, or EONs. EONs are designed to specifically bind to the target (single stranded) RNA and mimic the double stranded structure that normally attracts an enzyme called Adenosine Deaminase Acting on RNA (ADAR). An EON can attract ADAR to a specific location in an RNA to make an A-to-I edit. This technology can be used to correct an RNA with a disease-causing mutation back to a normal RNA, or change a protein so that it will have a new function that helps prevent or treat disease.

The company’s current pipeline includes discovery stage programs addressing areas of high unmet medical need: AX-0810 for Cholestatic Diseases targeting: NTCP, and AX-1412 for Cardiovascular Disease targeting: B4GALT1.

See also

References
  1. Let's Chat About...ProQR's work in treatments for inherited retinal disease. Hope in Focus. YouTube. 31 January 2022. Retrieved 11 February 2022.
  2. "Axiomer RNA editing technology – A novel way to repair RNA". www.proqr.com. Retrieved 14 June 2023.
  3. Weisman, Robert (20 January 2018). "Henri Termeer isn't exactly settling into retirement". The Boston Globe. Boston Globe Media Partners, LLC. Retrieved 11 February 2022.
  4. McBride, Ryan (10 May 2013). "Henri Termeer rediscovers the 'Genzyme feel' in world of biotech startups". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  5. Carroll, John (9 September 2014). "Termeer-backed ProQR lays out terms for $75M IPO". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  6. Taylor, Nick Paul (13 September 2017). "ProQR spins out CNS assets to form RNA rare disease biotech". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  7. Rodríguez Fernández, Clara (12 March 2018). "A Dutch Company on the Quest Against Cystic Fibrosis". LaBiotech.eu. Retrieved 11 February 2022.
  8. Rodriguez Fernandez, Clara (14 February 2017). "New Dutch Spin Out will Treat Stroke with RNA Therapy". LaBiotech.eu. Retrieved 11 February 2022.
  9. Adams, Ben (8 September 2021). "Eli Lilly follows up MiNA pact with a 2nd $1B-plus RNA collab, this time with ProQR". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  10. Newman, Christoper (22 December 2022). "Lilly pays $75M to widen RNA editing deal with ProQR". BiopharmaDive. Archived from the original on 22 December 2022. Retrieved 23 December 2022.
  11. "ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs | ProQR Therapeutics". www.proqr.com. Retrieved 14 June 2023.
  12. Van Zuidam, Joanne (22 September 2017). "The Art of Editing RNA". Drug Discovery Trends. Retrieved 4 April 2018.
  13. Dale, Alex (13 February 2018). "ProQR Tackles Blindness Caused by a Rare". LaBiotech.eu. Retrieved 11 February 2022.
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