Driving Gene Therapy Beyond Rare Disease Will Define The Next Era Of CGT

By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

At last month’s 2026 @Philly Cell and Gene Therapy Annual Conference, one message came through clearly for me. The future of cell and gene therapy will not be defined only by ultra rare disease success stories. It will be defined by whether companies can responsibly scale these technologies into broader patient populations while preserving safety, durability, manufacturing quality, and commercial viability. That is why Ocugen’s Executive Vice President of Commercial and Business Development Abhishek Gupta’s presentation stood out.

Gupta offered one of the clearest examples yet of how the industry is attempting to move gene therapy from niche indications into larger ophthalmology markets with millions of patients. His remarks reflected where the CGT industry is headed next and what companies must solve to succeed there.

Ocugen’s strategy centers on ophthalmology, specifically retinal diseases, where the company believes localized delivery and measurable clinical outcomes create an attractive path for gene therapy expansion. But what made Gupta’s presentation particularly relevant for the broader CGT field was not simply the pipeline. It was the framework behind it.

“The technology is validated,” he said. “We know that it works.”

That confidence is increasingly shared across the industry. The question now is no longer whether gene therapy can work in humans. The next challenge is whether companies can scale these therapies into larger diseases without repeating the commercialization and manufacturing difficulties that have slowed parts of the sector.

Why Ophthalmology May Become a Proving Ground

Gupta repeatedly emphasized why ophthalmology offers unique advantages for advancing gene therapy beyond ultra rare disease applications. “This is localized delivery, not systemic,” he explained. “The eye is immune privileged. It’s compartmentalized. Most importantly, the outcomes are measurable.”

That combination matters. One of the biggest hurdles in expanding gene therapy into broader indications is balancing efficacy with safety in much larger populations. In ophthalmology, companies can directly monitor disease progression and treatment effects while limiting systemic exposure.

Ocugen’s pipeline reflects this strategy. The company is advancing programs in retinitis pigmentosa, Stargardt disease, and geographic atrophy, with ambitions to secure three BLA submissions across three years.

The progression from rare inherited retinal diseases toward geographic atrophy, a much larger indication affecting millions of patients, represents a notable transition point for the field. According to Gupta, this expansion is intentional and years in the making. “Where we netted out a while ago is that we really targeted geographic atrophy as a key area where we thought our initial technology could be applied to larger diseases,” he said.

The Industry’s Manufacturing Reality Check

One of the most important parts of Gupta’s remarks involved manufacturing and scalability. While many conference discussions focused on clinical data, Gupta highlighted operational realities that continue to define whether CGT companies succeed commercially.

“CMC is hard enough for rare diseases, but now you have to solve it for larger diseases,” he said. “The question is always just because you can do it, just because you’ve got great science, can you supply it?”

That comment captures one of the defining pressures facing the industry. Manufacturing remains one of the largest bottlenecks in gene therapy development. Scaling production from hundreds of patients to potentially millions fundamentally changes the economic and operational requirements. Gupta stressed that manufacturing strategy must begin early. “You have to address those manufacturing components really, really early,” he said. “The earlier thinking really pays off in the long run.”

For CGT companies hoping to move into larger indications, this may be one of the most actionable lessons from Ocugen’s experience. Scientific innovation alone will not determine winners in the next phase of gene therapy. Companies must also demonstrate reproducibility, scalability, and long-term supply readiness.

A Shift Toward Gene-Agnostic Platforms

Another major theme from Gupta’s presentation was Ocugen’s focus on “modifier gene therapy,” a gene agnostic platform approach designed to address diseases driven by multiple mutations or pathways.

Traditional gene therapies have largely focused on replacing or correcting a single defective gene. But Gupta argued that approach becomes increasingly difficult in diseases involving hundreds or thousands of mutations. “In retinitis pigmentosa, there are many mutations implicated,” he said. “Creating one gene therapy at a time for 100 mutations is perhaps not the most feasible way to go.”

Instead, Ocugen’s platform uses a viral vector to deliver a nuclear hormone receptor intended to regulate broader disease pathways rather than targeting a single mutation.

For the CGT field, this represents an important strategic evolution. As companies pursue larger patient populations, mutation-specific therapies may become commercially and operationally unsustainable in some disease categories. Platform technologies capable of addressing multiple genetic variants at once could become increasingly attractive to regulators, payers, and clinicians.

Durability Remains the Commercial Differentiator

Throughout the presentation, Gupta repeatedly returned to one concept that continues to dominate discussions across gene therapy development. Durability.

“The ability to have durable responses is incredible,” he said.

For ophthalmology specifically, Ocugen is positioning one-time retinal injections as a meaningful alternative to chronic injection schedules currently required in diseases like geographic atrophy.

Gupta pointed to high discontinuation rates with current standards of care. “About 40 percent of patients drop off the current standard of care in a year and a half,” he said.

That treatment burden creates a major opening for durable gene therapies if companies can prove long term efficacy and safety.

Importantly, Gupta highlighted that Ocugen now has three-year durability data in retinitis pigmentosa. In a market increasingly scrutinized for long-term outcomes, that kind of follow up data becomes essential not only for regulators but also for physicians, investors, and payers.

The Broader Implications for CGT

What made Gupta’s presentation particularly compelling was that it reflected the maturation of the entire CGT industry. Several years ago, many companies focused almost exclusively on proving gene therapy could work in ultra rare diseases. Today, the conversation is changing. The field is beginning to ask harder questions around scalability, patient access, manufacturing economics, durability, and commercial sustainability.

Ocugen’s pipeline illustrates both the opportunity and the complexity of that transition. “The business case, the scientific case, the clinical case has to be really robust,” Gupta said.

That statement may ultimately define the next decade of CGT development. The companies that succeed will likely be those capable of integrating science, manufacturing, regulatory strategy, clinical execution, and commercialization from the beginning rather than treating them as separate stages.

For the broader industry, Gupta’s presentation served as a reminder that the next era of gene therapy will require a fundamentally different operating model than the first. The science may already be validated. Now the industry must prove it can scale.