Proqr Therapeutics

Today ProQR has announced the transaction for Thea to acquire Sepofarsen and Ultevursen Ophthalmic Assets has been completed.

Press release here: https://bit.ly/46TZf0S

“Following previous announcements related to this transaction, we are pleased to share that we have now completed the divestment of the sepofarsen and ultevursen programs to Théa, who will continue the development of these potentially transformational therapies for patients with LCA10 and Usher Syndrome” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR.

ProQR has received an initial payment of €8M and may be eligible for up to €165M in further development, regulatory, and commercial earn-out payments upon related achieved milestones, as well as double-digit royalties based on commercial sales in the US and EU.

Earlier this week, our Chief Scientific Officer Gerard Platenburg penned an article in BioSpace, explaining recent developments in and its potential to treat both rare genetic disorders and common conditions for patients in need.

Read more about his perspective on the strength and potential of , and why it’s widely considered the next generation of promising medicine here:
https://www.biospace.com/article/interest-in-rna-editing-accelerates-as-therapies-approach-the-clinic-/

If you want to learn more about Axiomer, visit our dedicated webpage: www.proqr.com/science/axiomer-technology or check out our short informative videos: www.youtube.com/watch?v=2KmGkQF24cQ&t=15s

Opinion: Interest in RNA Editing Accelerates as Therapies Approach the Clinic | BioSpace The next frontier in RNA therapies, RNA editing has the potential to treat both genetic and common disorders, and the technology is rapidly expanding beyond the liver.

Today we have provided an update that our previously announced agreement to divest the company’s late-stage ophthalmic assets to Théa has been terminated.

Read more:

ProQR Therapeutics Provides Update on Ophthalmic Assets ProQR is a clinical stage biotech company based in the Netherlands that discovers and develops unique RNA-based therapies for severe genetic disorders.

ICYMI. Last Saturday Leiden hosted the first .

We were so proud of the dedicated ProQRians who showed our commitment to divesity, equity, and inclusion in our organization.

Pride Leiden wants to give visibility to all people who, for whatever reason, feel different.



http://prideleiden.nl

We are excited to be a sponsor of the upcoming this coming saturday!

ProQR will be the 3rd boat in the canal parade.

Pride Leiden wants to give visibility to all people who, for whatever reason, feel different.

www.prideleiden.nl/

ICYMI. Earlier this year we caught up with returning ProQRian Rene Beukema. Check out our community blog on the ProQR website.

Catching up with returning ProQRian René Beukema In this personal interview, we speak to ProQR’s Chief Corporate Development Officer & General Counsel, René Beukema on his return to ProQR.

After additional analyses from our Illuminate trial and an in-depth strategic review, we will focus on exploring a development path for selected ophthalmology programs and accelerate development of our Axiomer RNA editing technology.

Read the community update: https://www.proqr.com/community-stories-and-news/proqr-update-and-additional-sepofarsen-illuminate-trial-analyses

ProQR update and additional sepofarsen Illuminate trial analyses ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.

Happy Rare Disease Day! In this series' last video researcher Jeroen explains his passion to solve the many problems he encounters when designing new therapies for rare eye conditions.

“Problem solving here means helping somebody else”

https://www.youtube.com/watch?v=HlaFSaAHLFc

"I work on designing and optimizing new therapies" - Jeroen van de Giessen Researcher at ProQR Jeroen works in our innovation department where they discover new potential therapies for rare eye conditions. In this process he encounters a legion of prob...

Nani from Regulatory Affairs advises our product teams on the best strategies to bring a new drug to patients. “Rare Disease Day is an opportunity for us to learn and implement that into our drug development.”

https://www.youtube.com/watch?v=P_WC5m2smok

"In rare diseases patients are the experts" - Nanyamka Braham from Regulatory Affairs at ProQR In the Regulatory Affairs team Nani advises our product teams on the best strategies to bring a new drug to patients. In this video she explains what moved h...

At ProQR we are dedicated to changing lives through developing novel RNA therapies for rare genetic diseases.

Within the Medical Affairs team Agathe tries to better understand how rare disease impacts people’s lives and the unmet medical needs that exist. “I decided to work in rare disease because I think no-one should be left aside”

https://www.youtube.com/watch?v=55zA0qu5r30&list=PLSB-fSXJfpfm6qibPn7YO4dbzR-StcRJS&index=7&t=5s

"Towards removing all barriers in rare diseases" - Agathe Plichta from Medical Affairs at ProQR In the Medical Affairs team at ProQR Therapeutics Agathe tries to better understand how rare conditions affect people's lives and by knowing the unmet medica...

Less than a week to go until Rare Disease Day 2022! Rare disease day is observed on the last day of February because it is the rarest day of the year. We join the community in raising awareness and trying to generate change.

Follow us this week for a series of videos providing insights into drug development for genetic conditions and what inspires ProQRians to work in rare disease.

Learn more: https://www.proqr.com/community-stories-and-news/rare-disease-day-2022

Rare Disease Day 2022 On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.

From all ProQRians we wish you happy holidays and an especially bright 2022!

We hope to stay closely connected with the retina and ophthalmology community in the new year and we anticipate exciting milestones in developing treatments for genetic eye disease.

From all at ProQR Therapeutics, happy thanksgiving everyone!

Happy International Women's Day!

Led by our Diversity, Equity and Inclusion team ProQR celebrates this day with wearing purple and sharing what we choose to challenge and call out gender bias and inequality.

Daniel remains a passionate advocate for and instills this across all . From the work in our to our teams we are striving to always act in the interest of patients.

Learn more: https://www.proqr.com/founding-story

ProQR founding story When our CEO, Daniel de Boer, knew his newborn son had an incurable rare disease, he founded ProQR to help people like his son. Learn more.

Today is ! At ProQR we are proud to be a part of the community and our teams are working tirelessly to help better the lives of those living with .

In our Leiden labs dedicated scientists from our teams are taking our RNA from the petri dish to the .

Learn more about for : https://www.proqr.com/RNA-therapy

RNA therapy Find out how RNA therapy works, how it differs from gene therapy and why we use it to treat genetic diseases that are currently untreatable.

Reposting this fun video that we did last year in celebration of . Rare is Strong, Rare is Proud, Rare is Many!

https://www.youtube.com/watch?v=PDC26ySBXY0

Rare Disease Day 2020 at ProQR Therapeutics ProQRians embracing the Rare Disease Day colors & messages! Read more about this important awareness event on the last day of February: https://rarediseaseda...

11th February is International Day of Women and Girls in Science. With the majority of working in science and being female (60%), this is something to celebrate. A big thanks to all the talented, smart and inspiring !