LEIDEN, Netherlands & CAMBRIDGE, Mass. — ProQR Therapeutics NV, a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced a collaboration with the Rett Syndrome Research Trust (RSRT) that will focus on the design and development of editing oligonucleotides (EONs) using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (MECP2) and correcting mutations of interest.
“The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Axiomer has the potential to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome. This collaboration marks an important step in accelerating the development of Axiomer in CNS, one of our initial areas of strategic focus along with liver-originated diseases.”
“ProQR’s leading RNA editing technology platform, coupled with their deep expertise in developing oligonucleotide therapies will offer an exciting pathway to develop new therapies for Rett Syndrome. RNA approaches can offer several advantages, including, for example, that because of their small size they do not require viral delivery,” said Bob Deans, RSRT’s Chief Technology Officer and Head of Research. “Building on work RSRT has been funding in the laboratory of Dr. Peter Beal at UC Davis, we are excited to now also work closely with ProQR to advance RNA editing as a potential therapeutic approach for Rett syndrome.”
RSRT awarded ProQR approximately $1 million as a research grant for the initial phase of the project, which will encompass editing oligonucleotide design and optimization, including evaluation in in vivo models for editing efficacy and MECP2 protein recovery. It is the intent of the partnership to be continued by an expanded co-funding arrangement following the initial discovery work. The co-funding of the next phase of the collaboration would enable clinical development of an Axiomer-based therapeutic for Rett syndrome MECP2.
About Axiomer™
ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
About Rett Syndrome
Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
Investor contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
[email protected]
Media contact:
Robert Stanislaro
FTI Consulting
T: +1 212 850 5657
[email protected]