Skylark Bio has dosed the first patient in the SONIX Phase I/II clinical trial evaluating SKY-GJB2, its investigational inner-ear-targeted gene therapy for GJB2-mediated hearing loss. “Dosing the first patient in SONIX brings us one step closer to a future where children born with genetic hearing loss have new… Read More
The latest episode of the DDW Highlights Podcast is now available to listen to below. DDW’s Bruno Quinney narrates five key stories of the previous week to keep DDW subscribers up-to-date on the latest industry news. Last week, the UK made headlines for both the investment it has attracted and… Read More
News round-up for 27 April – 1 May by Bruno Quinney, Content Team at DDW. This week, the UK has made headlines for both the investment it has attracted and the clinical trial reforms it has rolled out. Elsewhere, the world’s first antimalarial drug for newborns could soon address a critical unmet… Read More
The US Food and Drug Administration (FDA) has granted accelerated approval for Regeneron Pharmaceuticals’ Otarmeni (lunsotogene parvec-cwha), the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher programme. Otarmeni is an adeno-associated virus vector-based gene therapy indicated for the treatment… Read More
Eli Lilly and Seamless Therapeutics are teaming up to develop and commercialise programmable recombinase-based treatments targeting hearing loss indications, using Seamless’ proprietary recombinase platform. The company’s technology performs large, precise DNA insertions in any target gene sequence, and operates independent of the cell’s natural DNA repair… Read More
New gene therapy trial results have shown dramatic and sustained improvements in hearing and speech perception in children with profound genetic hearing loss. The updated data for Regeneron Pharmaceuticals’ investigational gene therapy DB-OTO for profound genetic hearing loss due to variants of the otoferlin (OTOF) gene were… Read More
Lineage Cell Therapeutics has entered a research collaboration with and through an investment from William Demant Invest (WDI), the holding company for William Demant Foundation’s investment activities and a majority shareholder of hearing healthcare company, Demant. The parties will jointly advance Lineage’s auditory neuronal cell transplant, ReSonance… Read More
Eli Lilly has entered into an agreement to develop and commercialise novel RNA-editing therapies using Rznomics’ proprietary trans-splicing ribozyme platform. Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The collaboration focuses on the discovery and development of… Read More
Pedmarqsi (sodium thiosulfate solution for infusion) has become the first treatment to be made available within NHS Scotland for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients up to 18 years of age with localised, non-metastatic, solid tumours. The decision follows that of… Read More
Regeneron Pharmaceuticals has revealed updated data for the investigational gene therapy DB-OTO from the Phase I/II CHORD trial in 12 children who have profound genetic hearing loss due to variants of the otoferlin (OTOF) gene. These include 72-week results showing speech and development progress… Read More
Five children who were born with congenital hearing loss can hear in both ears after taking part in a gene therapy trial at Fudan University in Shanghai, China. Within weeks of receiving the therapy, the children could locate the sources of sounds, and recognised speech in… Read More
The investigational gene therapy DB-OTO improved hearing to normal levels in a child (dosed at 11 months of age) within 24 weeks, according to trial data presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference. Initial hearing improvements have also been observed… Read More
In the first case of its kind, gene therapy has restored hearing in a patient in the United States. Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 Phase I/II study participant. AK-OTOF is a gene… Read More
Biotechnology company Sensorion has enrolled the first patient in its Phase IIa Proof of Concept clinical trial of SENS-401 (Arazasetron) in patients suffering from cisplatin-induced ototoxicity. Cisplatin and other platinum-based compounds are essential chemotherapeutic agents for many cancers. A serious side effect of these therapies is ototoxicity,… Read More
Biotechnology company Rinri Therapeutics has been awarded an Innovation Passport by the UK Medicines and Healthcare products Regulatory Agency’s (MHRA) for its cell therapy targeting a form of hearing loss. The Innovation Passport, which is awarded through the MHRA’s Innovative Licensing and Access Pathway (ILAP), will enable… Read More
