Lizards, zebrafish, salamanders and tritons can regrow a tail, a fin, or even an entire limb after amputation. Cut a planarian into pieces, and you will end up with a bunch of them. Researchers at the National Institute of Biological Sciences in Beijing have discovered a genetic switch linked to vitamin A. After activating this pathway, they managed to regenerate the ear pinna of a mouse, an animal that previously lacked this ability.
In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.
Sound Pharmaceuticals Inc.’s lead candidate, the anti-inflammatory SPI-1005 (ebselen), has produced positive pivotal phase III study data in treating Meniere’s disease, an inner-ear disorder that can lead to vertigo and deafness.
The primary-endpoint win by Sensorion SA in a phase II proof-of-concept study with SENS-401 (arazasetron) in hearing loss made public March 11 brought renewed interest in the space, where a number of players are advancing gene therapies. The story marches on, with Montpellier, France-based Sensorion due to discuss the product July 13 at the International Conference on Cochlear Implants and Other Implantable Technologies in Vancouver, British Columbia.
Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.
RNA therapy developer Proqr Therapeutics NV’s phase I/II study of adults with Usher syndrome and non-syndromic retinitis pigmentosa met all its key objectives, prompting the company to plan two parallel pivotal phase II/III studies that could start by year-end. The company wasted no time as it has already discussed next steps with the FDA to support the therapy’s registration “as soon as possible,” Aniz Girach, Proqr’s chief medical officer, told investors during a March 24 investor call.
A phase IIa study of Frequency Therapeutics Inc.’s lead candidate in treating hearing loss showed it fared no better than placebo, but the company said its faith in the candidate is bolstered by the belief the study was hampered by trial design issues and by data from another study.
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