Cell therapy weekly: promising long-term results for gene therapy targeting inherited deafness
This week: Ray Therapeutics (CA, USA) secured US$125 million in Series B funding for its vision restoration platform, and a gene therapy for inherited deafness demonstrated promising long-term results. Plus, Kyverna Therapeutics (CA, USA) reported positive outcomes in treating stiff person syndrome with its CAR-T therapy, miv-cel.
The news highlights:
- Series B funding secured to advance vision restoration therapies
- Promising long-term results for gene therapy targeting inherited deafness
- Encouraging CAR-T therapy results for autoimmune disease treatment
Series B funding secured to advance vision restoration therapies
Ray Therapeutics, a clinical-stage biopharmaceutical company, has successfully closed a US$125 million Series B financing round to advance its vision restoration platform for treating retinal degenerative diseases. The company’s lead gene therapy, RTx-015, is currently under evaluation for retinitis pigmentosa. Delivered via a single intravitreal injection, RTx-015 aims to restore functional vision by introducing a bioengineered, highly light-sensitive protein to targeted retinal cells in patients with advanced visual impairment.
“Following our recent receipt of Regenerative Medicine Advanced Therapy Designation from the US Food and Drug Administration for our lead clinical program, we are deeply appreciative of the tremendous enthusiasm for this financing among leading investors,” commented Ray Therapeutic’s CEO and Co-Founder Paul Bresege. “These investors’ proven ability to identify value-creating biopharmaceutical companies reinforces our position at the forefront of vision restoration innovation. I am grateful to our existing investors for their continued support as well as our new Series B investors who were excited to add [Ray Therapeutics] to their portfolios. Together, we share a commitment to life-changing treatments for patients living with blinding diseases.”
The funding round was led by Janus Henderson Investors (London, UK), with additional participation from new investors, including Adage Capital Management (MA, USA), Franklin Templeton, Invus, and Marshall Wace (all London, UK). The round also received strong follow-on support from existing investors.
Promising long-term results for gene therapy targeting inherited deafness
A new international study co-led by investigators from Mass General Brigham (MA, USA) and the Eye & ENT Hospital of Fudan University (Shanhghai, China) demonstrated the success of a gene therapy, AAV1-hOTOF, for treating autosomal recessive deafness 9 (DFNB9), a rare genetic form of hearing loss caused by mutations in the OTOF gene.
The study found no serious side effects, and 90% of participants experienced hearing improvement within weeks, leading to better speech recognition and language development. Younger children and those with healthier inner ears showed the most significant improvements, while participants treated in both ears achieved higher language and speech scores compared to those treated in one ear. Among the three adults treated, two showed hearing recovery.
“These multicenter trial results validate the effectiveness of our OTOF gene therapy,” said Yilai Shu, a professor from Eye & ENT Hospital of Fudan University, who led the study. “The procedure can be broadly implemented in hospital settings, ensuring consistent delivery for a larger patient population.”
The researchers plan to continue long-term follow-up and hope to start a US trial in the future.
“These results show that restoring hearing is possible even after years of deafness,” continued Shu. “We are now working to expand this approach to other genetic causes of hearing loss.”
Encouraging CAR-T therapy results for autoimmune disease treatment
Kyverna Therapeutics presented positive results from its registrational trial of miv-cel for stiff person syndrome, a rare and progressive neurologic autoimmune disease characterized by muscle stiffness and painful muscle spasms, at the American Academy of Neurology annual meeting (18–22 April 2026; IL, USA). The trial, KYSA-8, showed that a single dose of the CAR-T therapy miv-cel led to rapid, statistically significant and clinically meaningful improvements across all primary and secondary endpoints at 16 weeks. Most patients regained physical and mental function, and all were able to discontinue chronic immunotherapies – outcomes not previously observed in stiff person syndrome.
“The results from our KYSA-8 registrational trial mark a defining moment for Kyverna, and more importantly, for patients living with stiff person syndrome,” said Warner Biddle, CEO of Kyverna Therapeutics. “We see compelling evidence that a one-time therapy can reset the immune system, reverse the course of disease, and free patients from lifelong treatment burden. With no approved therapies, we believe miv-cel could redefine the treatment paradigm for this debilitating, progressive disease. We are preparing our [Biologics License Application] submission for this initial indication, and the data strengthen our confidence in miv-cel’s therapeutic potential in myasthenia gravis, as well as other neurologic autoimmune diseases.”
