ProQR and RSRT Expand Collaboration to Build on RNA Editing Momentum
Of all the novel genetic medicine modalities that we are advancing, RNA editing is the closest to the clinic. RSRT funding is making it possible for ProQR to advance their Rett program.
Earlier this morning ProQR, a biotech company located in The Netherlands, announced a significant expansion of an existing collaboration with RSRT to advance RNA Editing therapeutics for Rett syndrome. The original collaboration that kicked off earlier this year generated exciting data that inspired this expansion.
The entire RNA Editing industry has been invigorated recently with the early success of a clinical study sponsored by Wave Therapeutics for liver disease. We recently highlighted this landmark with a blog article.
We're very enthusiastic about RNA Editing for several key reasons. RNA editing involves correcting a Rett mutation at the level of messenger RNA rather than DNA. A major benefit is that due to their small size, these RNAs can be injected and taken up like a small molecule drug and does not require viral or non-viral delivery. The stability of modified small RNAs is high, with half-lives of 6 to 9 months suggesting dosing would entail intrathecal injection twice a year.
Not having to deal with AAVs removes the complexity and cost of manufacturing which will translate to better patient access and reduces risk due to potential toxicity. Importantly, it gives patients more options since they can theoretically access a virally delivered genetic medicine and an RNA Editing therapeutic in much the same way as SMA (Spinal muscular atrophy) patients can receive zolgensma and spinraza.
Another advantage of RNA Editing is that correcting the mutation ensures the cells will make the right amounts of MECP2 in every cell type.
Of all the novel modalities we are advancing, RNA Editing is closest to the clinic. Our funding to date has generated robust proof of concept editing data in patient cell lines showing a high degree of editing. The new funding will support IND-enabling studies in rodent and non-human primate studies involving dose-ranging with repeated dosing.
MECP2 mutations that can be edited via RNA editing include G>A mutations, Nonsense Mutations, and C-terminal Deletions. Collectively, these mutations account for 40% of all Rett cases.
ProQR will be using the R270X mutation to show success and then quickly take on the other mutations that can be addressed with this approach.
Our deep held philosophy is that no individual with Rett will be left behind. Some of our approaches are “generic” in that they can address all mutations, while others are mutation-specific. We won't rest until every individual with Rett has genetic medicine options.
It's important to note that the award to ProQR is not a grant but an investment. If the therapeutics are commercialized payments will flow back to RSRT, all of which will be reinvested in research.
Our initial commitment to ProQR is $2.65 Million, which will support the program up to clinical trials and, if the data continues to be encouraging, a further commitment of $5.5 Million for the clinical trial. ProQR is matching RSRT’s investment.
This collaboration highlights an important aspect of RSRT’s role in the development of genetic medicines for Rett. As outlined in our Roadmap to Cures, Rett is not low-hanging fruit, and companies are unlikely to initiate programs on their own. The funding we provide de-risks programs by generating critical data, which can incentivize companies to fully bring programs in-house and support them with their own funding.
We cannot thank our families and donors who support us enough. This is your money at work on behalf of our children!
