Each year hundreds of children are born legally blind because of a genetic disorder that degrades their retinas. Though some can distinguish between light and dark early on, a defect in their AIPL1 gene leaves most without sight by the time they turn four. The disorder has long been considered irreversible.
But that’s no longer true. A new gene therapy from publicly traded company MeiraGTx has managed to do something unprecedented: It’s given eyesight to 11 children who were born blind.
Surgeons at Great Ormond Street Hospital and the Evelina London Children’s Hospital injected the retinas of kids born with the AIPL1 defect with an MeiraGTx therapy that introduces a correct version of the gene, which causes their bodies to produce the mechanisms that enable sight. Within weeks they were able to see for the first time. Now, years later they all are capable of reading, coloring and playing like other children with the aid of glasses.
“These were blind kids and now they run around and find rooms. They can read letters. They can read numbers,” said Zandy Forbes, 60, the founder and CEO of MeiraGTx. The therapy is one of several the company is working on to treat eye-related disorders and other conditions such as Parkinson’s, obesity and xerostomia.
In the initial study, which was published in The Lancet last week, four children with the disorder were given the therapy in one eye each. Within a month, the treated eye began to see again. It could identify colors, shapes, and letters and follow moving objects. There were no significant side effects. Encouraged by this success, the hospitals gave the therapy to seven more children, this time in both eyes. All seven had their eyesight restored. Given the strength of these initial results, MeiraGTx is pursuing regulatory approval in the United Kingdom and the United States.
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Forbes, who is not related to the Forbes media family, started MeiraGTx in 2015 after leaving her role as an SVP of commercial operations at pharmaceutical company Kadmon (which has since been acquired by Sanofi). The company raised a total of about $110 million in venture capital from investors including University College London’s Technology Fund, ID Fund, and Perceptive Advisors before going public in 2018 with an IPO that raised $75 million. It reported about $12 million in revenue for the first nine months of 2024, a figure that Forbes expects to increase as soon as its first therapy is approved by regulators.
In late 2023 MeiraGTx sold a license for another gene therapy to Johnson & Johnson in a deal worth up to $415 million and received a milestone payment of $50 million in 2024. Pharma giant Sanofi also made a $30 million investment in MeiraGTx in 2023 in exchange for a right of first negotiation for several of its drug candidates, bringing its total investment in the company to $60 million. The company has about $120 million in cash which gives it runway through 2026.
Gene therapies often require complicated procedures to deliver them. Forbes has made it a point to focus on the development of ones that are both easy to administer and treat both genetic diseases and other maladies.
To that end, the company has focused on low-dose treatments, which lowers the risk of side effects. Like most gene therapies, its treatments are delivered to patients using specially modified viruses called “adeno-associated viruses” (AAVs), essentially a type of cold virus that has the infectious part of the virus removed and replaced with whatever medicine needs to be delivered.
But unlike other gene therapy companies, MeiraGTx is vertically integrated, and has built commercial-ready manufacturing facilities so it doesn’t have to rely on contractors to make its gene therapies. That has enabled the company to rapidly complete clinical studies, said Forbes, who named the company for the Hebrew word for “giving light.”
After its IPO, where shares were priced at $15 per share, MeiraGTx’s stock soared. Like many early-stage biotech stocks, it peaked during the pandemic years–almost doubling its IPO price. But it also suffered the same post-pandemic slump impacting a number of early-stage biotechs. At market close on Tuesday, it was trading at $6.68, giving it a market cap of about $522 million, which is up about 7% year to date.
Earlier this year, investment bank Chardan named MeiraGTx as one of its top 25 biotech stock picks for the year. In a research note published about the announcement, analyst Daniil Gaaulin at investment bank Chardan wrote, “We believe the strength of the data, which we find transformative … substantially de-risk the path to the U.K. approval and lay a foundation for favorable regulatory discussions with the FDA.”
Gaaulin has set a price target for the stock at $36. Other analysts have also rated the stock a buy and consider it underpriced because they expect that its AIPL1 treatment will be approved soon.
In addition to the APL1 treatment, MeiraGTx is developing several other gene therapies for inherited retinal disorders, which have all progressed to human testing. It’s working on a treatment for head and neck cancer patients whose salivary glands were damaged by radiation therapy, where they are injected with the genes to produce saliva again. It’s also developing a proprietary way to more precisely deliver drugs like gene therapies and peptide therapeutics (which include GLP-1s like Ozempic). These drugs are typically injected into the body or infused via IV, flooding the bloodstream with a higher dose of drugs which can increase the risks of side effects. Precise delivery allows for lower doses, which mitigates risk and has the potential to reduce cost.
One major company focus is gene therapy to treat Parkinson’s. Here, its treatment stimulates production of a neurotransmitter called Gamma-aminobutyric acid (GABA) in the brain. Low levels of GABA are thought to be one cause of the disease. In a small study the company published last year that has not yet been peer reviewed, a high dose of the therapy improved motor function and quality of life in patients who received it.
With this data in hand, Forbes said, her company will be pursuing a larger clinical trial later this year.
Correction: This article has been revised since publication to reflect the correct surgery locations and timing of a milestone payment.