#RNAediting
🧬 AMBER: RNA editing game-changer! Precise C-to-U conversion without DNA damage. 21% efficiency in mouse models = huge potential for genetic disease treatment 🔬 #GenomicMedicine #RNAEditing #Biotech https://emmecola.github.io/genomics-daily
November 7, 2025 at 6:35 AM
The replay of our Investor and Analyst Event is now available: bit.ly/43PqFWS

Thanks to Professor Verkade for joining us! We are delighted that AX-0810 progressing to the clinic.

#Axiomer #RNAEditing $PRQR
November 3, 2025 at 5:02 PM
Join us in 60 mins for our virtual Investor and Analyst Event with ProQR Management and KOL Prof. Henkjan Verkade, MD, PhD (UMC Groningen).

Register for the event here: bit.ly/3Lb7GzE

#Axiomer #RNAEditing $PRQR
November 3, 2025 at 2:00 PM
October 30, 2025 at 11:31 AM
Join us Nov 3 at 10:00 ET for our virtual Investor and Analyst Event with the ProQR Management and KOL Prof. Henkjan Verkade, MD, PhD (UMC Groningen).

Register for the event here: bit.ly/3Lb7GzE

#Axiomer #RNAEditing
October 27, 2025 at 1:37 PM
October marks #RettSyndromeAwarenessMonth, a time to shine a light on Rett syndrome.

At ProQR, we are proud to partner with the Rett Syndrome Research Trust developing #Axiomer #RNAEditing oligonucleotides to target genetic variants that cause Rett syndrome.
October 22, 2025 at 1:05 PM
We’re excited to share that our CTA for the Phase 1 study of AX-0810 in healthy volunteers has been authorized!

Join our virtual Investor and Analyst Event Nov 3, 10:00 ET.

For more details, check out the PR: bit.ly/3LjRCLX

#Axiomer #RNAEditing
October 20, 2025 at 12:48 PM
David, Senior Scientist at ProQR, plays a key role in advancing cell biology research while also contributing to team leadership and workplace culture.

Meet the rest of our team at: bit.ly/4nbVZWR

#Axiomer #RNAEditing #TeamProQR
October 15, 2025 at 9:47 AM
🚨 New paper alert! 🚨

Our paper entitled: "Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3" is out now on Journal of Biological Chemistry!

www.jbc.org/article/S002...

#AlternativeSplicing #RNAediting

🧵 👇
Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3
Alternative splicing and adenosine-to-inosine (A-to-I) RNA editing are post-transcriptional mechanisms that promote proteomic diversity in the brain. The impact of RNA editing on brain physiology is u...
www.jbc.org
October 14, 2025 at 2:34 PM
🔗 In-depth analysis of the RNA editing landscape in intracranial aneurysms and its potential role in alternative splicing. Computational and Structural Biotechnology Journal, DOI: doi.org/10.1016/j.cs...

📚 CSBJ: www.csbj.org

#RNAediting #Neuroscience #Genomics #PrecisionMedicine #BrainHealth
September 30, 2025 at 4:41 AM
Genetic editing holds promise to treat incurable diseases, but #CRISPR sometimes does more harm than good. @UCSanDiego.bsky.social and @Yale.edu researchers report a new, potentially safer method: small nuclear #RNAediting.
Small Nuclear RNA Base Editing a Safer Alternative to CRISPR, UC San Diego Researchers Find
Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale…
buff.ly
September 18, 2025 at 3:04 PM
RNAediting working well though.
September 3, 2025 at 1:35 PM
Adenosine-to-inosine editing of miR-200b-3p is associated with the progression of high-grade serous ovarian cancer

👉 buff.ly/FMrZvtO

#HGSOC #miR200 #RNAediting
September 1, 2025 at 8:33 AM
Lemelinda pairs her passion for RNA biology with research, protocol development & biosafety support, while also fostering a strong community at ProQR through our Coffee & Collaboration Café.

Meet more of the ProQR team at: bit.ly/45ZFtmq

#Axiomer #RNAEditing #TeamProQR
August 26, 2025 at 9:36 AM
Today we announced our Second Quarter 2025 Operating and Financial Results. Read the press release here: bit.ly/4fwEU7Y
$PRQR #RNAEditing #Axiomer
August 7, 2025 at 11:48 AM
Genetic changes in an RNA-editing enzyme reduce editing of a brain receptor gene, leading to abnormal receptor function, neuronal overactivity, seizures, and developmental delay.
#RNAediting #Neurodevelopment bit.ly/3TGUN1i
July 25, 2025 at 6:31 PM
Great to connect with the local community at the Dutch Medicines Summer Afternoon!

Our VP Axiomer Science, Lenka van Sint Fiet, demonstrated how #Axiomer is driving progress toward potentially transformative therapies.

#RNAEditing #DutchBioScienceWeek
July 25, 2025 at 11:42 AM
As R&D Team Manager at ProQR, Lorenza drives scientific collaboration and project planning while also championing inclusivity as part of our DEI team.

Meet the ProQR team at: bit.ly/4m4oZzs

#Axiomer #RNAEditing #TeamProQR
July 22, 2025 at 8:45 AM
We are happy to announce the submission of a CTA to the EMA to initiate a Phase 1 clinical trial in healthy volunteers for AX-0810 targeting NTCP.

Check out the press release for more details: bit.ly/4ljFzuQ

#Axiomer #RNAEditing #BiotechNews
June 26, 2025 at 8:40 PM
Rapid and Cost-Effective Digital Quantification of RNA Editing and Maturation in Organelle Transcripts
Hua, Z.
Paper
Details
#RNAEditing #OrganelleTranscripts #DigitalQuantification
June 25, 2025 at 9:01 AM
At ProQR, we’re proud to foster a culture where everyone feels safe to be themselves. To celebrate #Pride, our DEI team hosted a rainbow themed coffee & collaboration event with multicoloured cakes and a movie night featuring an LGBTQIA+ film and discussion.

#Inclusivity #RNAEditing #Axiomer
June 12, 2025 at 7:33 AM
This week we held our biannual Axiomer Summit event – a day of vibrant discussions, insightful presentations, and an engaging poster session focused on our science. Thanks to our Scientific Advisory Board and Board members for joining this exciting exchange!

#RNAEditing #Axiomer #Biotech
June 6, 2025 at 11:56 AM
#Regeneron is such a cool #genetics-based biotech, but they should also become expert in modalities, not just licensing them. #CRISPR and #RNAediting are ideally suited and they should have them in-house. Prices are cheap, too!
June 2, 2025 at 3:04 PM
One good thing in the 'One Big Beautiful Bill' is that drugs with more than 1 #orphan indications will be excluded from IRA negotiations. This is relevant for #RNAi #RNAediting and #CRISPR targeting things like TTR, LDL-R, ApoB.
May 22, 2025 at 8:30 PM
#Alnylam trades at a 50x market cap of #Intellia directly exposed to CRISPR for many of its high-value liver indications.

How on earth do they not have strategic option to #CRISPR, even through a 50:50-type partnership with a leading CRISPR Co if not in-house?

#RNAediting similar situation.
May 18, 2025 at 6:23 PM