Articles from Oak Hill Bio

Oak Hill Bio, a biotechnology company focused on developing life-changing therapies for people with rare diseases, today announced the publication in Nature Medicine of the results of the rugonersen Phase 1 TANGELO study in 1 to 12 year old children with Angelman syndrome.1 Oak Hill Bio recently obtained exclusive rights to rugonersen and plans to initiate a Phase 3 study in patients with Angelman syndrome in early 2026. The results published today (The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial, Hipp et al., Nature Medicine, DOI 10.1038/s41591-025-03784-7) showed adverse event rates for rugonersen consistent with other antisense oligonucleotides, the route of administration, and the patient population. The results also showed that treatment with rugonersen led to dose-dependent partial normalization of pathological brain activity on a pharmacodynamic biomarker of brain function, electroencephalogram (EEG) delta power, and improvements on multiple exploratory endpoints measuring the core symptoms of Angelman syndrome as compared to natural history. The safety and tolerability profile, as well as the encouraging pharmacodynamic and exploratory clinical effects observed in TANGELO, provide strong support for further testing of rugonersen for the treatment of Angelman syndrome.

Oak Hill Bio, a biotechnology company focused on acquiring and developing life-changing rare disease therapeutics, has entered into an exclusive license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) to obtain global rights for rugonersen (RO7248824). Rugonersen is an ASO designed as a potential best-in-class treatment for individuals with Angelman syndrome, a rare, genetic neurodevelopmental condition for which there is no approved disease-modifying treatment. Oak Hill plans to initiate a Phase 3 study in early 2026.

Oak Hill Bio, a clinical-stage neonatology and rare disease therapeutics company, and Chiesi, an international, research-focused biopharmaceutical company (Chiesi Group), announced that the first European patient has been enrolled in a Phase 2b clinical study, restarted in May, to assess the efficacy and safety of OHB-607, an investigational drug candidate being developed to prevent complications of extremely premature birth, including bronchopulmonary dysplasia (BPD), a serious condition for which there are no approved therapies.

Oak Hill Bio, a clinical-stage neonatology and rare disease therapeutics company, today announced the promotion of Josh Distler, J.D. to Chief Executive Officer. Additionally, the Company has appointed Sharon Morriss, Ph.D. as Chief Operating Officer and promoted Ike Greenstein, MBA to Chief Business Officer. Oak Hill also welcomed Jiaping Gu, Hussein Al-Mossawi, and Kim Davis to its Board of Directors.

Oak Hill Bio, a clinical-stage neonatology and rare disease therapeutics company, and Chiesi, an international, research-focused biopharmaceutical group (Chiesi Group), announced that the first patient has been enrolled in a resumed Phase 2b clinical study to assess the efficacy and safety of OHB-607, an investigational drug candidate being developed to treat complications of extremely premature birth, including bronchopulmonary dysplasia (BPD), a serious condition for which there are no approved therapies.

Chiesi Farmaceutici S.p.A (“Chiesi Group”), an international, research-focused biopharmaceuticals and healthcare group, and Oak Hill Bio, a clinical-stage neonatology and rare disease therapeutics company, today announced the execution of a License and Development Agreement to develop, manufacture, and commercialize OHB-607, an investigational drug candidate being developed to treat complications of extremely premature birth.

Oak Hill Bio (“Oak Hill”), a clinical-stage neonatology and rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced that members of its senior leadership team will participate in one-on-one meetings with investors at the upcoming Citi 17th Annual BioPharma Conference taking place on September 7-8, 2022, in Boston, MA.

Oak Hill Bio (“Oak Hill”), a clinical-stage neonatology and rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced that members of its senior leadership team will participate in virtual one-on-one meetings with investors at the upcoming 2022 Wells Fargo Private Biotech Symposium taking place on June 1, 2022.

Oak Hill Bio (“Oak Hill”), a clinical-stage neonatology and rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced that the Company sponsored a symposium at the 10th Annual International Conference on Clinical Neonatology that took place from May 21-23, 2022 in Turin, Italy. The conference session highlighted potential indications for OHB-607, the Company’s proprietary, recombinant human version of insulin-like growth factor 1 (IGF-1) and its main binding protein, IGFBP-3.

Oak Hill Bio (“Oak Hill”), a clinical-stage neonatology and rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced that Tauhid Ali, Ph.D. has been appointed the Company’s first Chief Executive Officer. Dr. Ali has more than 25 years of experience in the global biopharmaceutical industry, with broad management and leadership experience in translational research, drug development, and corporate strategy.

Oak Hill Bio (“Oak Hill”), a clinical-stage neonatology and rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced that members of its senior leadership team will participate in the upcoming 2022 RBC Capital Markets Global Healthcare Conference taking place May 17-18, 2022 in New York City. Oak Hill Bio management will deliver a corporate presentation highlighting the Company’s clinical programs and plans for the future on Wednesday, May 18, 2022 at 10:00 a.m. ET.

Oak Hill Bio (“Oak Hill”), a clinical-stage neonatology and rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced the formation of the Company’s Board of Directors, which brings together a team of experienced leaders with collective expertise spanning financing, business operations, drug discovery, clinical development and biopharmaceutical business development. The board is chaired by Edwin Moses, Ph.D., former Chairman and Chief Executive Officer of Ablynx.

Oak Hill Bio (“Oak Hill”), a clinical-stage rare disease therapeutics company developing life-changing medicines for extremely preterm infants and patients suffering from rare autoimmune diseases, today announced the Company’s launch and plans to advance a pipeline of promising clinical and preclinical investigational therapeutics acquired and licensed from Takeda Pharmaceutical Company Limited (“Takeda”).