#ProQR
#RNAediting patent app by #ProQR targeting gain-of-function in MC4R in the #CNS in #obesity.

patentscope.wipo.int/search/en/de...
March 25, 2025 at 9:31 PM
ProQR welcomes Chris Hart as Chief Data & AI Officer and Thomas Wolf as Strategic AI Advisor to the Board. Their appointments support our strategy to apply AI across discovery and development. AX-0811, the first AI-discovered ProQR program is now in clinical trials.

#Axiomer #RNAEditing
September 23, 2026 at 12:27 PM
#ProQR impressive preclinical AX-0810 cholestatic disease #ASGCT25 abstract. Clinical candidate shows >50% #RNAediting.

Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
May 13, 2025 at 9:49 AM
#ProQR said yesterday they are still in late stages of experiments ahead of CTA filing and data still on track for 2025.

So I expect CTA to slip into Q3. Today important presentation on what hope is the clinical candidate at #asgct25. #RNAediting

www.biotechtv.com/post/proqr-a...
ASGCT 2025: The Co-Founder of ProQR discusses the company's Axiomer RNA base editing technology - which will see a CTA filed this quarter and data by the end of the year
ProQR Co-Founder & CSO Gerard Platenburg describes the company's RNA editing platform and the journey he has seen this field go through over time. Plus, updates on programs for cholestatic diseases, R...
www.biotechtv.com
May 16, 2025 at 10:04 AM
#ProQR another nice gain-of-function #RNAediting patent app, this time inspired by a human genetic variant in the #reelin gene conferring 'extreme resistance' to #Alzheimer's.
April 3, 2025 at 6:17 AM
#ProQR here is the van Herk letter. Makes important points, including violation of Dutch securities laws regarding Director term limits.

Annual Meeting next week.

www.sec.gov/Archives/edg...
www.sec.gov
May 30, 2025 at 10:05 AM
Very satisfying to see progress on #RNAediting efficiencies. Also >50% in #RettSyndrome program. Just 2 years ago, 1-3% were top hits without ADAR overexpression.

#ProQR
May 13, 2025 at 10:00 AM
$PRQR re-iterates Q2 CTA guidance for cholestatic disease program w/ first biomarker data in Q4.

Overall moving more into CNS. Cash into H2 2027, $LLY still w/ opt-in rights for 5 additional targets.

www.proqr.com/press-releas...
ProQR Announces Year End 2024 Operating and Financial Results
ProQR is a clinical stage biotech company based in the Netherlands that discovers and develops unique RNA-based therapies for severe genetic disorders.
www.proqr.com
March 13, 2025 at 11:54 AM
#ADARediting $PRQR quarterly update. All you need to know:

NTCP cholestatic disease candidate on track for Q2 CTA filing and Q4 readout (healthy volunteer, biomarker)

Cash runway into mid-2027.

finance.yahoo.com/news/proqr-a...
ProQR Announces First Quarter 2025 Operating and Financial Results
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened ...
finance.yahoo.com
May 8, 2025 at 1:42 PM
#ProQR unusual selling volume (2% of outstanding). 1.8 million shares traded so far today. Will it bottom today?

Q2 critical CTA/IND filing guidance with first human data expected in Q4 and cash runway into mid 2027...not worried here, it's in the company's hands.
March 27, 2025 at 7:14 PM
#ProQR presents progress on ADAR Therapeutics at GRC:

Really like the robust ADAR activity in #CNS. Single-dose in rat/NHP --> ICV in rats: high editing in brain; intrathecal in NHP: high in spinal cord.

Imagine systemic admin--> high editing everywhere in CNS.
www.proqr.com/files/2025-0...
March 25, 2025 at 7:42 AM
#ProQR had hoped to see NHP editing efficiencies for new AX-0810 #cholestatic disease candidate, but largely preclinical data with older (less potent candidate).

Guidance for CTA remains Q2 (though I am skeptical they can meet it) and topline data in Q4.
www.proqr.com/files/2025-0...
May 16, 2025 at 9:58 PM
A little help on article view and translation. What are your thoughts on how this relates to van Herk holding over 11% of proqr?
May 29, 2025 at 12:44 PM
Nice 80% #RNAediting efficiency in patient-derived cells at therapeutically relevant #oligonucleotide concentrations. If #ProQR can take advantage of #FDA platform pathway, #Rett opportunity very exciting.

#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
May 17, 2025 at 9:52 AM
#ProQR votes are out. deBoer (86.3%) + Shannon (80.7%) re-appointed as Directors. Should encourage corp governance discussion.

Only 78.5% of votes cast were in favor of authorizing Board to issue shares. Not sure the 'no' are aware that we are in the biotech business?

www.sec.gov/Archives/edg...
www.sec.gov
June 3, 2025 at 8:37 PM
Today we recognize the 11th anniversary of International Day of Women and Girls in Science! We caught up with Cristina Lopez Lopez, MD, PhD, CMO at ProQR, who shared her message for aspiring #WomenInScience.

#IDWGS2026 #Feb11 #EveryVoiceInScience
February 11, 2026 at 7:35 AM
ProQR forms advisory board, partnership with Ginkgo as part of AI strategy
seekingalpha.com
April 8, 2026 at 1:47 PM
$PRQR ProQR adds 8.1 million in funding to advance Rett drug into Phase I trial

www.proqr.com/press-releas...
ProQR Therapeutics Announces $8.1 Million in New Funding from Rett Syndrome Research Trust to Expand RNA Editing Collaboration
ProQR is a clinical stage biotech company based in the Netherlands that discovers and develops unique RNA-based therapies for severe genetic disorders.
www.proqr.com
December 11, 2024 at 12:46 PM
Bridging the gap from Lab to Business.

These scientific scale-ups are hiring PhD talent:
- Mission Zero in London (Climate Tech)
- Michabo in Birmingham (Toxicology)
- ProQR Therapeutics in Leiden (Biotechnology)
- Proteros Biostructures in Munich (Biotechnology)

(Continuing in thread)

#PhD
March 9, 2025 at 2:19 PM
Van Herk stapt naar rechter vanwege conflict met biotechbedrijf Proqr
Van Herk stapt naar rechter vanwege conflict met biotechbedrijf Proqr
De Rotterdamse investeerder eist een onderzoek naar het bestuursbeleid bij de beursgenoteerde onderneming uit Leiden.Proqr verwacht dat het verzoek wordt afgewezen.
fd.nl
August 3, 2026 at 3:02 PM
At what point will #Chinese biotechs buy up Western #biotechs with critical tech and IP, incl #PrimeMedicine and #ProQR?
April 10, 2025 at 5:19 PM
August 8, 2024 at 6:50 PM
I must say #ProQR is on probation right now. Having delayed clinical entry in this biotech environment was daring.

My demands:

1) It has to be for a substantially better candidate, show NHP data along with CTA news

2) CTA Q2 guidance must be met.
May 30, 2025 at 8:10 AM