Rugonersen

CAMBRIDGE, Mass., Oct. 06, 2026 (GLOBE NEWSWIRE) -- OHB Pediatrics Ltd d/b/a Oak Hill Bio, a clinical-stage rare disease therapeutics company, today announced the appointment of Joerg Hipp, PhD as Chief Translational Science Officer. Dr. Hipp was integral to the development of Oak Hill Bio’s lead asset, rugonersen, and brings extensive expertise in neuroscience, neurodevelopmental disorders, translational science, and biomarkers. “We are thrilled to welcome Joerg to Oak Hill Bio at such an exciting time for our company,” said Josh Distler, Chief Executive Officer of Oak Hill Bio. “Joerg was an integral member of the team that led rugonersen's initial clinical development. He is an outstanding scientist and a leading expert on the use of electroencephalogram (EEG) as a biomarker. His decision to join Oak Hill is a reflection of his tremendous dedication to and faith in rugonersen. I look forward to working with him as we execute on our Phase 3 trial of rugonersen, with the goal of demonstrating that high levels of UBE3A restoration can translate into improvement of pathological brain activity and a meaningful clinical benefit for individuals living with Angelman syndrome.” “I am honored to join the Oak Hill Bio team at such an important stage of the company’s journey to deliver a treatment for Angelman syndrome,” said Joerg Hipp, Chief Translational Science Officer of Oak Hill Bio. “Over the past decade, I’ve had the privilege of working on Angelman syndrome and the rugonersen program, which allowed me to become highly familiar with rugonersen’s mechanism of action, the electrophysiological measurements that provide an objective window onto abnormal brain function, and the clinical assessments to evaluate improvement in Angelman syndrome. I look forward to continuing this work and supporting the pivotal clinical development of rugonersen.” Dr. Hipp brings over 15 years of experience in neuroscience across leading research institutes and the biopharmaceutical industry. Dr. Hipp joins Oak Hill Bio from Roche, where he was a biomarker and experimental medicine leader responsible for biomarkers across their neurodevelopmental and neuromuscular disorder franchise and led the Electrophysiology & Analytics group. Dr. Hipp authored approximately 70 peer-reviewed articles (https://orcid.org/0000-0002-7875-2988) in the fields of neuroscience, electrophysiology, neurodevelopmental disorders, and clinical trials with more than 10 articles focusing on Angelman syndrome including as lead author of the research article summarizing the results from rugonersen’s Phase 1 TANGELO trial, published last year in Nature Medicine (https://www-nature-com.libproxy1.nus.edu.sg/articles/s41591-025-03784-7). Dr. Hipp earned an M.S. in Physics from the University of Innsbruck, Austria, and a Dr. sc. nat. in Neuroscience from ETH Zurich, Switzerland. About Oak Hill Bio Oak Hill Bio is a clinical-stage biotechnology company focused on acquiring and developing promising therapeutics for rare diseases with significant unmet needs that have been deprioritized by pharmaceutical companies. The company's lead program is rugonersen (OHB-724), an investigational antisense oligonucleotide (ASO) in Phase 3 clinical development as a potential best-in-class treatment for Angelman syndrome, a devastating neurodevelopmental disorder with no approved disease-modifying therapies. Oak Hill Bio is the trading name for OHB Pediatrics Ltd. It was formed in 2024 as a subsidiary of Oak Hill Bio Holdings (formerly known as Oak Hill Bio Ltd). Learn more at oakhillbio.com. About Angelman syndromeAngelman syndrome (AS) is a serious rare genetic neurodevelopmental disorder which causes severe mental and physical impairment and affects approximately 15,000 individuals in each of the US and the EU5, with an estimated incidence of 1 in 12,000 to 20,000 live births. AS is characterized by global developmental delay, intellectual disability, epilepsy (90% of cases before age 3 years) with an atypical underlying electroencephalogram (EEG), ataxia, tremor, hyperactivity, limited speech, and sleep dysregulation. Symptoms emerge during infancy and persist throughout life. Deletions and mutations in the maternal ubiquitin protein ligase E3A (UBE3A) allele cause Angelman syndrome; the paternal UBE3A allele is epigenetically silenced by a long non-coding antisense RNA (UBE3A-ATS) in neurons. UBE3A is required for normal brain development and function. Failure to express UBE3A in central nervous system (CNS) neurons leads to a build-up of damaged or unwanted proteins that can impair normal neuronal development and function. About RugonersenRugonersen is an antisense oligonucleotide (ASO) designed to address the underlying disease biology of Angelman syndrome (AS) by specifically and potently binding the UBE3A-ATS transcript. Rugonersen binding is intended to trigger degradation of the UBE3A-ATS transcript in the CNS and therefore the unsilencing of the UBE3A paternal allele. Rugonersen allows neuronal expression of the paternal copy of the UBE3A gene, potentially improving neuronal function and development in AS patients. Rugonersen’s clinical and preclinical data are detailed in the following publications: Hipp, J.F., Bacino, C.A., Bird, L.M. et al. The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a Phase 1 trial. Nat Med (2025). https://doi-org.libproxy1.nus.edu.sg/10.1038/s41591-025-03784-7; Jagasia et al., Angelman syndrome patient-derived neuron screen leads to clinical ASO rugonersen targeting UBE3A-ATS with long-lasting effect in monkeys, Nucleic Acids Research (2025). https://doi-org.libproxy1.nus.edu.sg/10.1093/nar/gkaf851 Contacts Investors:John FrauncesLifeSci Advisorsjfraunces@lifesciadvisors.com Media:media@oakhillbio.com

The Phase III Aspire study of apazunersen (GTX-102) in Angelman syndrome failed to meet its primary endpoint, leaving the condition without an approved disease-modifying therapy. Ultragenyx Pharmaceutical (Nasdaq: RARE) reported that the study showed no difference between treated and control groups on the Bayley-4 cognitive raw score, the primary measure, nor on the Multidomain Responder Index (MDRI), a composite key secondary endpoint spanning five functional domains. As per the press release, the baseline characteristics of randomized patients were comparable and consistent with those enrolled in earlier studies, offering no obvious confound to explain the divergence from Phase I/II signals. The safety profile was consistent with prior experience. Apazunersen is an antisense oligonucleotide (ASO) delivered intrathecally, designed to suppress the UBE3A antisense transcript (UBE3A-AS) that silences the paternal copy of the UBE3A gene in neurons. In Angelman syndrome, the maternally inherited UBE3A allele is absent or non-functional; reactivating the paternal copy is the central therapeutic rationale. The drug held FDA Breakthrough Therapy Designation, Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation, as well as PRIME designation from the EMA — a regulatory profile that reflected the strength of the Phase I/II package. Angelman syndrom afffacts an estimated 60,000 patients in commercially accessible geographies, none of whom currently have an approved treatment for the underlying disease. Oak Hill Bio is developing rugonersen (RO7248824), originally an antisense program from Roche that targets the same UBE3A-AS mechanism. Following Phase I data from the TANGELO study, Oak Hill initiated a pivotal Phase III trial in July 2026. The Aspire failure will prompt scrutiny of whether the mechanistic approach is sound or whether the clinical endpoints chosen for this population are sufficiently sensitive to detect treatment effects within trial timeframes. Ultragenyx said it will evaluate the future of the apazunersen program in light of the result and implement expense reductions across its operations. CEO Emil Kakkis said the company would maintain focus on its commercial business, citing the recent approval of GENGLYCOS for glycogen storage disease type Ia and the potential approval of UX111 for Sanfilippo syndrome as near-term revenue drivers, with a stated path to profitability in 2027. Spot something wrong? Report an issue with this article