Ascend partners with industry to drive down gene therapy cost for better patient access

Gene therapies represent a trans­for­ma­tive approach in modern medicine. Unlike traditional drugs, they are able to treat and cure, or prevent, diseases by editing the body’s instruction manual, the DNA, to fix mistakes or add helpful new information. 

In December 2023, the Food and Drug Admin­is­tra­tion (FDA) approved two gene therapies, Casgevy and Lyfgenia, for sickle cell disease; regulators are expected to greenlight up to 17 more cell and gene therapies in 2024. Notably, Casgevy employs CRISPR/Cas9 technology, a first for an FDA-approved drug. CRISPR/Cas9 works by using a custom-designed RNA sequence to locate a specific DNA segment, where an enzyme like Cas9 then makes precise edits. The technology’s successful application in Casgevy underlines its potential to transform medical approaches to various genetic and non-genetic diseases.

Despite the growing number of gene therapies expected to be approved this year, a few obstacles stand in the way of widespread adoption. One involves the limitations of current drug production technology in meeting the specific demands of gene therapy. A second is the limited number of scientists, engineers, and quality analysts experienced in the rapidly evolving field of cell and gene therapy manufacturing.

DCVC Bio portfolio company Ascend is actively addressing these challenges. The company’s approach includes assembling a skilled team through strategic acqui­si­tions and driving innovation through the imple­men­ta­tion of advanced analytics and technology, essential for ensuring the purity and quality of gene therapies. Ascend is also moving toward achieving compliance with Good Manu­fac­turing Practice (GMP) standards, a set of FDA-defined benchmarks that ensure consistent quality in drug manu­fac­turing. Compliance with these regulations is mandatory for phar­ma­ceu­tical companies in the United States, and the FDA enforces GMP regulations and inspects phar­ma­ceu­tical manu­fac­turing facilities to ensure compliance. 

We spoke with Ascend CEO Mike Stella about the oppor­tu­ni­ties presented by the recent gene therapy approvals and how his company is positioning itself to capitalize on the coming revolution in personalized medicine.

DCVC: The FDA’s recent approvals of Casgevy and Lyfgenia mark a significant milestone in gene therapy for sickle cell disease. How do you envision gene and cell therapies impacting global healthcare in the next decade?

Stella: These therapies are a game-changer, offering highly effective and potentially curative treatments compared to the traditional standard of care. While there’s been some concern about their initial costs, they actually represent significant long-term savings by effectively managing chronic diseases. This will not only improve the quality of life globally but also reduce the healthcare cost burden. 

And it’s important to keep in mind that we’re just at the beginning. With a growing clinical pipeline and better regulatory under­standing, I expect a rapid advancement in these therapies. You’ll see a snowball effect over the next five years, where the timelines to approval will get shorter because everyone is building on a foundation of knowledge that grows expo­nen­tially year over year.

DCVC: How do these approvals specif­i­cally impact Ascend, and what role do you see the company playing in the broader landscape of cell-based gene therapies moving forward?

Stella: These FDA approvals are a major boon for the industry and, by extension, for companies like Ascend. They’re a validation of gene therapies and open up a broader pipeline for potential appli­ca­tions. We specialize in helping customers develop these trans­for­ma­tive therapies and bring them forward to the clinic. More funding for more diverse diseases means increased oppor­tu­ni­ties across the industry.

DCVC: With these new devel­op­ments in gene therapy, what’s next for Ascend? Can you share any upcoming projects or goals that reflect the company’s vision and strategy in the evolving landscape of gene and cell therapies? 

Stella: First, we’re focused on leveraging our expertise in innovation and process development. We’re making strides in improving product yields and quality in our facilities in Alameda, California, and Munich, Germany. This includes working on appli­ca­tions that could allow clients to reduce their manu­fac­turing scales signif­i­cantly, leading to cost savings. For instance, advance­ments in yields might enable a reduction from 500-liter to 200-liter production scales, giving the same amount of usable, high-quality product.

In Munich, we are also setting up a Good Manu­fac­turing Practice (GMP) laboratory. GMP is a system ensuring that products are consis­tently produced and controlled according to quality standards. It’s vital for our industry, partic­u­larly for clients needing product testing and release for clinical trials or commercial appli­ca­tions in Europe. We expect this laboratory to be operational by the middle of the second quarter of 2024.

Finally, on the manu­fac­turing front, we’re actively pursuing strategic acqui­si­tions in the United States. These acqui­si­tions will enhance our capa­bil­i­ties from clinical to commercial production. Our acquisition strategy emphasizes the talent and experience of the teams rather than just the facilities. We believe the industry needs experienced teams to drive production quality. We’re on track to finalize these acqui­si­tions by the end of the second quarter, further expanding our development, char­ac­ter­i­za­tion, and manufacturing capabilities.