Proqr Therapeutics

ProQR Therapeutics has submitted a Clinical Trial Application to the European Medicines Agency for AX-0810, marking the first clinical advancement of its Axiomer RNA editing platform.

• ProQR Therapeutics has appointed Dennis Hom as CFO and Dr. Cristina Lopez Lopez as CMO to support the advancement of its Axiomer™ RNA editing technology platform into clinical development. • Dennis Hom brings over 25 years of financial leadership experience, having raised more than $4.5 billion in capital and executed transactions exceeding $57 billion in value throughout his career. • Dr. Cristina Lopez Lopez contributes 20+ years of global R&D leadership with expertise in translational science, previously serving as Global Head of Neurodegeneration at Johnson & Johnson.

The global RNA therapy clinical trials market is projected to grow from $2.85 billion in 2024 to $4.16 billion by 2034, with a CAGR of 3.85%, driven by advancements in mRNA, siRNA, and antisense oligonucleotide-based therapies.

ProQR Therapeutics secures $8.1 million in additional funding from the Rett Syndrome Research Trust to advance AX-2402 into clinical trials.

• Beam Therapeutics reported a patient death in their BEAM-101 sickle cell disease trial, likely due to the conditioning regimen. • AstraZeneca scientists engineered PsCas9 for therapeutic genome editing in mouse liver, showing promise for hypercholesterolemia treatment. • YolTech Therapeutics' novel LNP system delivers base editor mRNA to bone marrow cells, activating foetal haemoglobin production for blood disorder treatment.

• Wave Life Sciences pioneered clinical RNA editing, treating alpha-1 antitrypsin deficiency (AATD) by correcting mRNA mutations, offering a novel therapeutic approach. • RNA editing, utilizing ADAR enzymes and guide RNAs, allows for temporary and reversible modifications, reducing risks associated with permanent DNA alterations. • Companies like Korro Bio and Ascidian Therapeutics are expanding RNA editing applications to target diseases like Parkinson’s, ABCA4 retinopathy, and certain cancers. • Challenges remain in RNA editing, including improving specificity, managing transient effects, and enhancing delivery methods for broader clinical applications.

• Wave Life Sciences reported positive proof-of-mechanism data from a single-dose trial of its RNA-editing therapy for lung and liver diseases, causing its stock to surge. • The trial data marks the earliest human data for any RNA editing therapy, validating the technology and exceeding analysts' expectations for early efficacy. • Following Wave Life's announcement, shares of other RNA-editing companies, including ProQR Therapeutics and Korro Bio, also experienced significant gains. • Wave Life plans to release data from a multi-dose trial of its RNA-editing therapy in 2025, further fueling optimism in the RNA-editing therapeutic space.

The Foundation Fighting Blindness's My Retina Tracker program offers free genetic testing and counseling for inherited retinal disease patients in the US, having enrolled over 6,000 participants since 2017.