Gene Therapy Advances, Major Biotech Investments, and Industry Trends from ISPE CaSA

Authored by Jake Greenwood, Vice President

On the heels of the industry’s biggest conferences, we can confidently say it is an exciting time to be in biopharma. With major breakthroughs and massive financing rounds, the sector is advancing at an incredible pace!

Regeneron’s Gene Therapy Breakthrough and Its Implications for Biomanufacturing

In an incredible feat, Regeneron’s DB-OTO gene therapy is showing promising early results in treating otoferlin-related deafness, restoring partial hearing in 10 out of 12 children. This is life changing for these children, many of whom were born completely deaf, and now, for the first time, they can hear their parents’ voices, respond to everyday sounds, and experience the world in an entirely new way.

As more companies work to bring similar therapies to market, the ability to scale production will be a key differentiator. With viral vector manufacturing already facing supply chain constraints, meeting demand for these therapies will require significant investment and strategic planning. Companies will need to decide whether to expand in-house production or partner with CDMOs to navigate the complexities of large-scale manufacturing and ensure a reliable supply.

In addition to hearing loss, gene therapy is also making headlines in oncology. A new gene therapy for glioblastoma, a highly aggressive brain cancer, is moving closer to clinical trials, backed by a major funding boost.

The Future of Gene Therapy Manufacturing – Glioblastoma Trial Funding

The California Institute for Regenerative Medicine has awarded $6 million to USC researchers to develop a first-of-its-kind gene therapy for glioblastoma.

Unlike traditional gene therapies this therapy uses an engineered AAV vector variant specifically designed to infect only cancer cells, minimizing off-target effects.

From a manufacturing perspective, this represents a new frontier in viral vector engineering. Developing vectors that selectively target diseased cells rather than relying on systemic delivery will require advanced process development and may have implications for the way we deliver these advanced therapies in the future.

The computational modeling being used to optimize drug delivery also signals a trend toward more integrated biomanufacturing approaches, combining AI-driven analytics with process development to improve efficiency and scalability.

While advancements in gene therapy are reshaping how we approach complex diseases like glioblastoma, the biotech industry as a whole is seeing a shift in how innovation is being funded.

Eikon’s $350M Fundraising Round and What It Signals for Biotech Investment

Eikon Therapeutics has secured a $350 million Series D, bringing its total funding to over $1.1 billion and reflecting a broader trend in biotech, where investors are prioritizing large, late-stage bets over early-stage plays.

More capital-intensive biologics, including novel immunotherapies and oncology treatments, require substantial investment in infrastructure. Eikon’s lead melanoma asset, in combination with Merck’s Keytruda, is in Phase 3 trials, meaning that commercial manufacturing strategies are already being developed.

The broader takeaway? Investors are still backing biotech, but increasingly more for companies that can demonstrate a clear path to commercialization. For manufacturers, this means an increasing emphasis on late-stage readiness, regulatory alignment, and scalable production strategies.

As investment pours into late-stage biotech, companies are scaling up their manufacturing capabilities to meet demand. Nowhere is this growth more evident than in North Carolina, where industry leaders recently met at the largest-ever ISPE CaSA Tech Show.

2025 ISPE CaSA Tech Show

The Project Farma team recently attended the CaSA Tech Show, where more than 3,000 attendees gathered to discuss the rapid expansion of biopharma manufacturing. The event highlighted North Carolina’s growing role as a major hub for biomanufacturing, with industry leaders sharing insights on the region’s continued investment and collaboration.

Site leadership from FUJIFILM Diosynth, Amgen, Thermo Fisher, and Eli Lilly participated in panels where they discussed their ongoing investments, highlighting the billions of dollars being poured into new manufacturing facilities.

One of the biggest themes discussed - why North Carolina has become such a hotspot for biopharma expansion.

Companies are drawn to the region not just for its business-friendly environment, but for its strong talent pipeline and infrastructure that supports large-scale biomanufacturing.

And as these facilities grow, collaboration between companies is becoming increasingly important, not just to drive innovation, but to ensure that the region remains a sustainable and competitive place to work.

Final Thoughts

The scale of investment we’re seeing from major biopharma players signals a fundamental shift toward strengthening domestic manufacturing capabilities.

With the rapid advancements in gene therapy, biologics, and next-gen therapeutics, the demand for cutting-edge facilities is only increasing. And if this year’s CaSA Tech Show proved anything, it’s that North Carolina is poised to be at the center of this growth.

About Project Farma

Project Farma, a Precision for Medicine company, specializes in providing comprehensive biomanufacturing strategies and execution services. We have successfully industrialized over 10 commercial cell, gene, and novel therapies, executed 60+ facility builds and capital expansions, and managed $6B+ in technical operations capital investments.

With expertise spanning startups, leading life science corporations, advanced therapy organizations, universities, hospitals, government agencies, and more, our team delivers proven technical operations and manufacturing solutions that shorten time-to-market, helping patients in need.

Visit our website to learn more about Project Farma and how Precision for Medicine’s integrated capabilities — across labs, clinical trials, manufacturing, and data intelligence— can help your organization bring therapies to life.