Ophthalmology is a booming niche, with investment flowing in, M&A up, and a burgeoning cast of biotechs – including the three European leaders profiled below – vying for a share of a global market set to top USD 93.7 billion by 2030

 

The field can already boast two bona-fide blockbusters: Roche’s Vabysmo (faricimab), which reached global sales of USD 5.3 billion in 2025, and Regeneron’s Eylea franchise, which generated USD 4.4 billion in the US alone as it successfully transitioned patients to its high-dose (8mg) formulation.

Beyond the current generation of injectables, non-invasive and gene-therapy approaches, such as those pioneered by Nanoscope and Astellas/Iveric Bio, are also poised to redefine the treatment landscape.

What is behind this meteoric rise? A growing awareness of preventive care is a crucial factor, as is age. As people live longer, the prevalence of age-related eye conditions like cataracts, macular degeneration, retinal diseases and glaucoma rises in step.

Lifestyle factors also play a part, with 50 percent of the world set to have myopia (nearsightedness) by 2050, largely due to more time spent in front of screens.

Already, around 90 percent of all young adults in Asia have some form of myopia. Increased inactivity is also giving rise to a diabetes epidemic, with several knock-on effects for eye health, including diabetic retinopathy (DR), diabetic macular edema (DME), cataracts and glaucoma.

 

GenSight Biologics

GenSight Biologics – a spin off from the Institut Vision translational research centre at the Quinze-Vingts National Ophthalmology Hospital in France – is looking to achieve a world-first in optogenetic therapy.

“We had been working on vision restoration in blind patients, harnessing gene therapy to introduce an opsin gene from green algae into these surviving retinal neurons,” recalls Serge Picaud, the institute’s Director. “One of the big unknowns had been whether the immune system would tolerate this algae-derived protein, which is completely foreign to the human body, but it did, and patients retained vision capabilities post-treatment.”

GenSight Biologics was created to advance this therapy – indicated for Leber Hereditary Optic Neuropathy (LHON), a devastating mitochondrial disorder that causes rapid and irreversible vision loss, often leading to blindness within a year – through clinical trials. The condition predominantly affects young men and currently has no approved curative treatment.

The therapy commanded positive early results and, in 2023, GenSight – by then publicly listed – had submitted an EMA marketing authorisation. However, perhaps speaking to the complexities of bringing gene therapies to market, weaknesses in GenSight’s dossier combined with manufacturing issues led to its withdrawal, a moment that CEO Laurence Rodriguez recalls as “incredibly difficult for all of us.” She subsequently resigned, as did several key colleagues.

Tempted back a few months later by the allure of GenSight’s science, Rodriguez has since overseen a strategic reset at the firm. A lean 15-person team is preparing for a new Phase III trial this year and has a new US CDMO partner to address previous manufacturing issues.

“Both EMA and FDA requested a new Phase III trial, even though 252 patients had already been treated in previous studies. Those trials, which compared one treated eye with the untreated control eye of the same patient, revealed an unexpected “contralateral effect,” whereby the therapeutic virus travelled via the optic nerve to the untreated eye. While scientifically fascinating, this biological phenomenon reduced the statistical difference between the two eyes and prevented a conclusive outcome, even though a meaningful improvement in patients’ vision was reported.”

“The upcoming Phase III trial, scheduled to begin in the second half of next year, will use a revised design with two independent patient groups – one receiving bilateral treatment with GS010 and the other a placebo – enabling a clearer, more statistically robust comparison.”

Rodriguez is bullish on the potential impact. “We remain humble but confident. The safety profile is excellent, the mechanism of action is well understood, and the dialogue we have maintained with both regulatory agencies has been extremely constructive. With these foundations, I am confident that we will be well-positioned to demonstrate efficacy and take a decisive step towards bringing LUMEVOQ to patients who have been waiting far too long for a therapeutic solution.”

There is even scope for attacking other indications via the company’s Mitochondrial Targeting Sequence (MTS) platform. “The potential applications of this technology extend well beyond LHON, offering a foundation for new therapies across multiple conditions,” exclaims Rodriguez.

 

Oculis

Led by industry veteran Riad Sherif, who cut his teeth at Sanofi and Novartis, Swiss-headquartered Oculis is hoping that its lead asset – currently in Phase III trials and the only topical “eye drop” therapy for DME in development globally – will come to represent a transformative breakthrough for large numbers of patients.

“DME affects approximately 35 million patients and represents the primary cause of blindness in the working-age adult population in the US,” explains Sherif. “However, the currently available treatment modalities rely exclusively on intraocular injections of VEGF inhibitors or steroids, creating significant barriers to early intervention and optimal patient outcomes.”

“Our topical delivery system addresses multiple clinical challenges simultaneously,” he continues. “First, it enables immediate treatment initiation upon diagnosis, potentially preventing irreversible vision loss. Second, it provides therapeutic options for the 40 percent of patients who demonstrate inadequate response to current injectable therapies.”

Oculis also has a dry-eye programme which is utilising biomarker-guided patient selection to improve the trial process. Even more ambitiously, it is moving into the complex field of brain health with its neuroprotection asset aimed initially at acute optic neuritis. Both assets are advancing into Phase II/III trials. “From the start, we aimed to build a diversified portfolio to avoid the binary risk typical in start-up,” explains Sherif.

“Rather than accepting existing treatment paradigms, we identify where current therapeutic options fail patients and engineer innovative alternatives. This philosophy is exemplified across our portfolio: OCS-01 represents the only topical “eye drop” therapy globally capable of treating DME without intraocular injection, while our precision medicine approach to dry eye treatment introduces biomarker-guided therapy selection—a first in ophthalmology.”

Sherif has high hopes that Oculis will eventually become a neuroscience platform company, addressing a potential market worth up to USD 50 billion. The company already boasts a strong financial position, with a cash runway through early 2028 and a logical strategy of direct US commercialisation combined with ex-US partnerships for international markets.

 

Tenpoint Therapeutics

Basel-based Tenpoint Therapeutics, meanwhile, is preparing for the 2026 launch of an eye drop product for presbyopia, an age-related condition which affects approximately two billion people worldwide. The company – the result of a 2024 merger between US-based Visus and UK-based Tenpoint – is in the process of tripling its workforce from 50 to 150 people to prepare for a “blockbuster-style” launch, led by a team with experience in over 30 product launches.

CEO Henric Bjarke explains the rationale behind this ambitious expansion. “Tenpoint has the only fixed-combination product under development in the presbyopia treatment category,” he notes. “Our miotic effect – the reduction of pupil size that enables improved near vision – represents the strongest demonstrated efficacy in the category. Beyond this primary mechanism, we have incorporated clinically meaningful endpoints that directly correlate with patient experience. Our Phase III data demonstrates significant improvements in reading speed at both three and six months, addressing the fundamental reason patients seek reading glasses.

He continues, “The inclusion of brimonidine in the formulation provides an additional aesthetic benefit through its eye-whitening properties, whilst the once-daily dosing regimen eliminates refrigeration requirements, significantly enhancing patient convenience and adherence potential.”

There are also big ambitions for how the company might evolve in the coming years. “We are constructing an ophthalmology company specifically focused on rejuvenating the ageing eye,” adds Bjarke. “Our programmes represent potential paradigm shifts… starting with presbyopia drops, moving to ‘cataract-reversing’ injections, and ending with cell therapy for geographic atrophy”

For Bjarke, it is the combination of commercial opportunity and unmet need, plus the speed at which innovations can be trialled and refined, that makes ophthalmology such a compelling investment proposition.

“The ophthalmology sector presents unique advantages for both innovation and commercialisation,” says Bjarke, “Unlike many therapeutic areas, ophthalmology offers relatively rapid feedback mechanisms for therapeutic efficacy, with anatomical and functional improvements often visible through diagnostic imaging and patient assessment.”

“Vision represents something universally relatable,” he concludes. “The prospect of vision loss resonates with everyone, creating both personal motivation and clear therapeutic impact”