ProQR

ProQR was founded in 2012 by chief executive officer (CEO) Daniel A. de Boer and co-founders Henri Termeer, Dinko Valerio and Gerard Platenburg.{{cite news|last1=Weisman|first1=Robert|title=Henri Termeer isn’t exactly settling into retirement|url=https://www.bostonglobe.com/business/2013/01/20/genzyme-henri-termeer-isn-exactly-settling-into-retirement/XTZKFMGnP2AewNMVkk1gbJ/story.html |work=The Boston Globe|publisher=Boston Globe Media Partners, LLC|date=20 January 2018|accessdate=11 February 2022}}{{cite news |last1=McBride |first1=Ryan |title=Henri Termeer rediscovers the 'Genzyme feel' in world of biotech startups |url=https://www.fiercebiotech.com/r-d/henri-termeer-rediscovers-genzyme-feel-world-of-biotech-startups |publisher=FierceBiotech. FierceMarkets Inc. |date=10 May 2013|accessdate=11 February 2022}}{{cite news |last1=Carroll |first1=John |title=Termeer-backed ProQR lays out terms for $75M IPO |url=https://www.fiercebiotech.com/biotech/termeer-backed-proqr-lays-out-terms-for-75m-ipo |publisher=FierceBiotech. FierceMarkets Inc.|date=9 September 2014|accessdate=11 February 2022}}{{cite news |last1=Taylor |first1=Nick Paul |title=ProQR spins out CNS assets to form RNA rare disease biotech |url=https://www.fiercebiotech.com/biotech/proqr-spins-out-cns-assets-to-form-rna-rare-disease-biotech |publisher=FierceBiotech. FierceMarkets Inc. |date=13 September 2017|accessdate=11 February 2022}}

The initial pipeline of the company was to develop small-molecule drugs or gene therapy that would treat cystic fibrosis (CF).{{cite news |last1=Rodríguez Fernández |first1=Clara |date=12 March 2018 |title=A Dutch Company on the Quest Against Cystic Fibrosis |publisher=LaBiotech.eu |url=https://www.labiotech.eu/interview/proqr-cystic-fibrosis-daniel-de-boer/#:~:text=Daniel%20de%20Boer%20founded%20ProQR,of%20people%20with%20cystic%20fibrosis |accessdate=11 February 2022}} 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. Subsequently, the company expanded its pipeline into treating other rare diseases, including diseases of the eye.

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.{{cite web|url=https://www.labiotech.eu/more-news/amylon-therapeutics-proqr-rna-therapy/|title=New Dutch Spin Out will Treat Stroke with RNA Therapy|first=Clara|last=Rodriguez Fernandez|publisher=LaBiotech.eu|date=14 February 2017|accessdate=11 February 2022}}

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 under which ProQR would receive US$50 million ($20 million upfront) and an equity investment of $30 million.{{cite web|url=https://www.fiercebiotech.com/biotech/eli-lilly-follows-up-mina-pact-a-second-1b-plus-rna-collab-time-proqr|title=Eli Lilly follows up MiNA pact with a 2nd $1B-plus RNA collab, this time with ProQR|first=Ben|last=Adams|publisher=FierceBiotech. FierceMarkets Inc. |date=8 September 2021|access-date=11 February 2022}} 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.{{Cite news |last=Newman |first=Christoper |date=22 December 2022 |title=Lilly pays $75M to widen RNA editing deal with ProQR |work=BiopharmaDive |url=https://www.biopharmadive.com/news/lilly-proqr-expand-rna-editing-partnership-deal/639408/ |url-status=live |access-date=23 December 2022 |archive-url=https://web.archive.org/web/20221222170715/https://www.biopharmadive.com/news/lilly-proqr-expand-rna-editing-partnership-deal/639408/ |archive-date=22 December 2022}}

In 2022, the company announced it would focus exclusively on advancing its RNA editing technology.{{Cite web |title=ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs|publisher=ProQR Therapeutics |url=https://www.proqr.com/press-releases/proqr-to-focus-exclusively-on-axiomer-rna-editing-technology-and-partner-ophthalmology-programs |access-date=2023-06-14 |website=www.proqr.com |language=en}}

ProQR is an RNA editing company targeting genetic diseases by focusing on making changes to the RNA.{{Cite news|url=https://www.drugdiscoverytrends.com/the-art-of-editing-rna/|title=The Art of Editing RNA|first=Joanne|last=Van Zuidam|date=22 September 2017|publisher=Drug Discovery Trends|access-date=4 April 2018|language=en}} The firm's RNA editing technology, called Axiomer can make targeted single nucleotide changes to RNA.{{cite news |last1=Dale |first1=Alex |date=13 February 2018 |title=ProQR Tackles Blindness Caused by a Rare |publisher=LaBiotech.eu |url=https://labiotech.eu/blindness-deafness-usher-syndrome-proqr/ |accessdate=11 February 2022}} 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 pipeline {{As of|2023|lc=y}} 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.{{cite web | title=Pipeline | publisher=ProQR Therapeutics | url=https://www.proqr.com/science/pipeline | access-date=6 December 2023}}

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