The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Initial results from Akouos Inc.’s phase I/II study showed that within 30 days of receiving AK-OTOF-101, pharmacologic hearing was restored to an 11-year-old who had profound hearing loss from birth.
Kriya Therapeutics Inc.’s unveiling of its new gene therapy program for thyroid eye disease (TED), KRIYA-586, added yet another player to the burgeoning space, where a handful of developers have reached the phase III stage.
E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.
N4 Pharma plc’s subsidiary Nanogenics Ltd. has signed a contract to start the formulation and sequence selection work to prepare its ECP-105 product for testing in preclinical studies.
Investigators from Proqr Therapeutics NV have tested their product QR-1011, an antisense oligonucleotide designed to correct splicing abnormalities within the ABCA4 gene that affect protein expression levels, in Stargardt disease.
Kriya Therapeutics Inc. has announced its gene therapy program for thyroid eye disease (TED), KRIYA-586. It is designed to be a one-time, adeno-associated virus (AAV) gene therapy that drives durable expression of a monoclonal antibody blocking the insulin-like growth factor 1 receptor (IGF-1R).
Diabetic retinopathy is the main diabetes complication affecting the eyes, and it leads to irreversible vision loss; oxidative stress and impairment of the retinal Muller cells are among the pathogenic mechanisms. Chinese researchers presented data on adiporon, an agonist of the adiponectin receptor protein 1 (ADPOR1) and ADIPOR2 that plays a crucial role in the metabolism of lipids and glucose, in preclinical mouse models of diabetes induced by streptozotocin.
Hangzhou Yirui Pharmaceutical Technology Co. Ltd. has described N-tetrazolyl aryl urea derivatives acting as bradykinin B1 receptor antagonists reported to be useful for the treatment of age-related macular degeneration, allergic conjunctivitis, pulmonary edema, neuralgia, infectious pneumonia, diabetic retinopathy, SARS-CoV-2 infection (COVID-19) and uveitis.
Putting into jeopardy what was on track to be the first approved therapy in Europe for geographic atrophy (GA), an advanced form of age-related macular degeneration that causes blindness, Apellis Pharmaceuticals Inc. learned of a negative trend vote by the EMA’s Committee for Medicinal Products for Human Use (CHMP) on its MAA for intravitreal pegcetacoplan.
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