Behind the Headlines Episode 12–Cell and Gene Therapies, New Genomics Tools, and Recent Partnerships

Behind the Headlines is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In this episode of Behind the Headlines, Brian Feth, CEO and founder of Xcell Biosciences; Thomas Heathman, chief commercial officer of Ori Biotech; and Matthew Lauber, Sr director of BioSeparations, Waters Corporation, discuss commercialization challenges in the cell and gene therapy (CGT) space and advances in new genomics tools from Roche, Illumina, and Eikon. In addition, the panelists review Biogen’s and Stoke Therapeutics’ collaboration for the development and commercialization of zorevunersen, a therapy for Dravet’s syndrome (1); Thermo Fisher Scientific’s purchase of Solventum’s purification and filtration business (2); and Eli Lilly’s $27 Billion plant expansion in the United States due to tax breaks extensions and tariffs threats

Pfizer said the discontinuation of its last remaining cell therapy program was due to several reasons, including “limited interest in gene therapies for the bleeding disorder…However, the company said it will redirect its time and resources towards Hympavzi, an injectable drug approved in October to prevent or reduce bleeding episodes in hemophilia A or B patients aged 12 years and older” (3). Our panel pointed out that a good amount of business process and commercialization rationale went into this decision. This coincides with the Carlysle groups purchase of a CGT Pioneer bluebird bio, for only $30 million (4), and illustrates investor wariness and strong commercial headwinds for the CGT field.

Recently, new genomics tools have been introduced into the market. Roche has unveiled a new class of next-generation sequencing with its novel sequencing by expansion technology (5), while at the same moment Illumina unveils first-of-its-kind spatial transcriptomics technology (6). In the same moment, Eikon has released novel single-molecule tracking technology, demonstrated through “clinical studies now operating in 28 countries, across five continents, we are accelerating the development of much-needed therapies while continuing to expand our research and development capabilities,” said Roger M. Perlmutter, MD, PhD, CEO and Board Chair of Eikon Therapeutics, in a press release (7).

References

1. Biogen. Biogen and Stoke Therapeutics Enter into Collaboration to Develop and Commercialize Zorevunersen for the Treatment of Dravet Syndrome, a Rare Genetic Epilepsy Associated with Refractory Seizures and Neurodevelopmental Impairments. Press Release. Feb 18, 2025.
2. Thomas, L. Thermo Fisher Strikes Deal for Solventum’s Filtration Unit,Wall Street Journal. Feb. 25, 2025.
3. Santhosh. C. Pfizer Stops Commercialization of Hemophilia Gene Therapy Beqvez, Reuters Business News. Feb. 21, 2025.
4. Carlyle. bluebird bio Announces Definitive Agreement to be Acquired by Carlyle and SK Capital. Press Release. Feb. 21, 2025.
5. Roche. Roche Unveils a New Class of Next-generation Sequencing with its Novel Sequencing by Expansion Technology. Press Release. Feb. 19, 2025.
6. Illumina. Illumina Unveils First-of-its-kind Spatial Transcriptomics Technology. Press Release, Feb. 19, 2025.
7. Genetic Engineering News. Eikon Therapeutics Raises $350 Million to Support Clinical Stage Programs, Pipeline Development. Genengnews.com. Feb 26, 2025.