An 11-year-old boy who received Eli Lilly’s experimental gene therapy for hearing loss last October showed signs of restored hearing 30 days after treatment, the pharma giant announced on Tuesday.

The therapy, dubbed AK-OTOF, is designed to deliver a functional copy of a gene called otoferlin. Without otoferlin, auditory signals received by the ear aren’t transmitted to the brain. Lilly estimates that 200,000 people worldwide experience hearing loss caused by mutations to otoferlin.

The company is testing AK-OTOF in a Phase I/II trial enrolling 14 patients, according to the clinical trials registry. The first child dosed, an 11-year-old born with hearing loss, achieved restored hearing “across all tested frequencies, achieving thresholds of 65 to 20 dB HL,” including results within normal hearing range at some frequencies, according to Lilly.

Lilly said it will release more details and additional data from a second patient at this year’s Association for Research in Otolaryngology in February. The company said both the therapy and the surgical procedure were well-tolerated with “no serious adverse events.”

AK-OTOF was the lead candidate at Akouos before it was bought by Eli Lilly in late 2022 for $487 million, plus $123 million in contingent value rights.

Regeneron read out results in October suggesting its rival gene therapy DB-OTO led to improved auditory responses in a child under 2 years old through 6 weeks. The therapy is in an early-stage study in the US, Spain and the UK.

Check out our AAV CDMO service to expedite your gene therapy research
About PackGene

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

Related News

How to make mRNA therapeutics safe from the start

The success of mRNA vaccines against COVID-19 has unleashed a flood of interest in using the technology to create more vaccines and treatments for everything from rare diseases and infections to cancer. But before new mRNA therapeutics are put to use, they need to be...

read more

Plasmids GMP Services

Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.

AAV GMP Services

Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
aav icon

Technology Platforms

PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.