Founder and Director General of Meiragtx Zandy Forbes
Omar Saval / Meiragtx
OfACH Year hundreds of children They are legally blind because of a genetic disorder that degrades their retina. Although some may distinguish between light and darkness early, a defect in their AIPL1 gene leaves mostly without looking at the time when four return. Disorder regulation has long been considered irreversible.
But this is no longer true. A new gene therapy from the publicly traded company Meiragtx has managed to do something unprecedented: glance at 11 children who were born blind.
Surgeons at Moorsfield Eye Hospital and University College London injected the retina of children born with AIPL1 defect with a meiragtx therapy that presents an accurate version of the gene, which causes their bodies to produce mechanisms. Within weeks they were able to see for the first time. Now, years later they are all capable of reading, dying and playing like other children with the help of glasses.
“These were blind children 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. Therapy is one of some that the company is working to treat eye -related disorders and other conditions such as Parkinson, overweight and Xerostomia.
In the initial study, which was published in Lancet Last week, four children with disorder were given therapy in one eye each. Within a month, the treated eye began to see again. Can identify colors, shapes and letters and follow the moving objects. There were no significant side effects. Encouraged by this success, hospitals gave therapy to seven other children, this time in both eyes. The seven had returned their gaze. Given the strength of these initial results, Meiragtx is following regulatory approval in the United Kingdom and the United States.
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Forbes, who has nothing to do with the Forbes Media family, began Meiragtx in 2015 after leaving its role as an SVP of trade operations at Kadmon pharmaceutical company (which has since been acquired by Sanafi). The company collected a total of about $ 110 million in investors from investors including the University of London University Technology Fund, ID and perceptual advisers before leaving in 2018 with an IPO that collected $ 75 million. She reported about $ 12 million in revenue for the first nine months of 2024, a figure that Forbes expects to grow as soon as her first therapy is approved by regulators.
At the end of 2023 Meiragtx sold a license for another gene therapy for Johnson & Johnson in a deal worth up to $ 415 million and expecting a $ 50 million stone payment when Stages 3’s stages. Pharma Sanafi’s giant also made a $ 30 million investment in Meiragtx in 2023 in exchange for a first negotiation right to some of its drug candidates, bringing his total investment in the company to $ 60 million. The company has about $ 120 million in cash it gives the runway by 2026.
Gene therapies often require complicated procedures to submit them. Forbes has made it a point to focus on the development of those that are easy to administer and treat both genetic diseases and other diseases.
To this end, the company is focused on low -dose treatments, which reduces the risk of side effects. Like most gene therapies, its treatments are delivered to patients using specially modified viruses called “AAVS viruses” (AAVS), essentially a cold virus that has the infectious portion of the virus removed and replaced with whatever medicine to be delivered.
But unlike other gene therapy companies, Meiragtx is vertically integrated, and has built ready -made commercial production facilities, so there is no need to rely on contractors to make its gene therapies. This has enabled the company to finish clinical studies rapidly, said Forbes, who named the company for the Jewish word for “giving light”.
After her IPO, where the shares were priced at $ 15 per share, Meiragtx’s shares increased. Like many biotechnical actions at an early stage, it peaked during the years of the Pandemia-Potheme, doubled its IPO price. But it also suffered the same post-fandemic decline affecting a number of early phase bioteks. In the near market on Tuesday, she was trading $ 6.68, giving her a $ 522 million market cap, which has grown about 7% so far.
Earlier this year, Chardan Investment Bank named Meiragtx as one of the top 25 elections of its biotechnical shares for the year. In a research note published on the announcement, analyst Daniil Gaaulin at the Chardan Investment Bank wrote, “We believe that the data force, which we find transformative … considerably de-rays the way to the adoption of the United Kingdom and lays a foundation for favorable regulatory discussions with FDA.”
Gaaulin has set a price target for stock at $ 36. Other analysts have also evaluated shares a purchase and consider it underestimated because they expect its AIPL1 treatment will be approved soon.
In addition to APL1 treatment, Meiragtx is developing some other gene therapy for inherited retina disorders, which have all progressed in human testing. Working working on a treatment for patients with head and neck cancer, whose salivary glands were damaged by radiation therapy, where they are injected with genes to produce saliva again. It is also developing a way of the owner to give more accurately drugs such as gene therapy and peptide therapeutics (which include GLP-1 as Ozepic). These medicines are typically injected into the body or injected through IV, flooding blood flow with a higher dose of drugs that can increase the risks of side effects. Accurate delivery allows lower doses, which mitigates the risk and has the potential to reduce the cost.
One major focus of the company is gene therapy to treat Parkinson. Here, its treatment stimulates the production of a neurotransmitter called gamma-aminobutric acid (GABA) in the brain. Low levels of GABA are thought to be a cause of the disease. In a small study, the company published last year that has not yet been reviewed by colleagues, a high dose of therapy improved motor function and quality of life in patients who received it.
With this data in hand, Forbes said, its company will follow greater clinical test later this year.
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