ProQR

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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/

ProQR Therapeutics NV (NASDAQ: PRQR) is a Dutch biotechnology company based in Leiden, the Netherlands. The company was funded in 2012 by chief executive officer (CEO) Daniel de Boer. It specializes in the development of RNA therapeutics for rare genetic diseases with an ophthalmologic application.

History[]

ProQR was founded in 2012 by chief executive officer (CEO) Daniel de Boer and co-founders Henri Termeer, Dinko Valerio and Gerard Platenburg.[1][2][3][4]

The initial pipeline of the company was to develop small-molecule drugs or gene therapy that would treat cystic fibrosis (CF).[5] 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.[5] Subsequently, the company expanded its pipeline into treating other rare diseases.[5]

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.[6]

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.[7]

Projects[]

The company’s current pipeline includes potential treatments for rare genetic diseases including Leber's congenital amaurosis, (LCA10) dystrophic epidermolysis bullosa, retinitis pigmentosa and Usher syndrome.[5][8][9]

ProQR is an ophthalmology company targeting genetic diseases by focusing on making changes to the RNA.[10] The firm's RNA editing technology, called Axiomer can make targeted single nucleotide changes to RNA.[9] The company's ultimate goal revolves around treating inherited retinal diseases by stopping and potentially reversing vision loss.[11] In addition to its headquarters in Leiden, the company is also active in Cambridge, Massachusetts.[11]

Drugs developed[]

One of the company’s lead candidates, QR-110, is being developed to treat LCA10.[12][13] The substance acts by binding the mutated RNA region which will allow the formation of a normal CEP290 protein.[14]

See also[]

References[]

  1. ^ 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.
  2. ^ McBride, Ryan (10 May 2013). "Henri Termeer rediscovers the 'Genzyme feel' in world of biotech startups". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  3. ^ Carroll, John (9 September 2014). "Termeer-backed ProQR lays out terms for $75M IPO". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  4. ^ Taylor, Nick Paul (13 September 2017). "ProQR spins out CNS assets to form RNA rare disease biotech". FierceBiotech. FierceMarkets Inc. Retrieved 11 February 2022.
  5. ^ a b c d Rodríguez Fernández, Clara (12 March 2018). "A Dutch Company on the Quest Against Cystic Fibrosis". LaBiotech.eu. Retrieved 11 February 2022.
  6. ^ Rodriguez Fernandez, Clara (14 February 2017). "New Dutch Spin Out will Treat Stroke with RNA Therapy". LaBiotech.eu. Retrieved 11 February 2022.
  7. ^ 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.
  8. ^ Rossi, Krista (25 May 2018). "Daniel de Boer, CEO of ProQR, Explains RNA Treatment, QR-313, for DEB". Rare Disease Report. Retrieved 11 February 2022.
  9. ^ a b Dale, Alex (13 February 2018). "ProQR Tackles Blindness Caused by a Rare". LaBiotech.eu. Retrieved 11 February 2022.
  10. ^ Van Zuidam, Joanne (22 September 2017). "The Art of Editing RNA". Drug Discovery Trends. Retrieved 4 April 2018.
  11. ^ a b Let's Chat About...ProQR's work in treatments for inherited retinal disease. Hope in Focus. YouTube. 31 January 2022. Retrieved 11 February 2022.
  12. ^ Mathias, Tamara (5 September 2018). "ProQR soars as childhood blindness drug succeeds in early trial". Reuters. Retrieved 4 April 2018.
  13. ^ Kilgore, Tomi (5 September 2018). "ProQR's stock rockets to pace all gainers after upbeat drug trial results". MarketWatch. Dow Jones & Company. Retrieved 4 April 2018.
  14. ^ Brown, Amy (6 September 2018). "Proqr hits the gas on blindness project". Evaluate.com. Retrieved 10 April 2018.

External links[]

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