Having spoken recently with CAR-T pioneer Bruce Levine, I found it interesting to hear him describe his International Society of Cell & Gene Therapies (ISCT) membership as a “kind of addiction”. Being involved in the ongoing development of one of humanity’s most advanced therapeutic technologies, and watching it evolve along the way, does seem like an irresistibly rewarding way to spend a career. Another pioneer in the field, Nobel Prize Laureate Jennifer Doudna, also continues to be involved in the evergreen development of CRISPR technologies through her involvement with Algen Biotechnologies, where she sits on the board of advisors.
A spinout of Doudna's lab at UC Berkeley, Algen’s co-founder and CEO Chun-Hao Huang has an academic history in genetic engineering and drug development. Using machine learning in the study of gene functions, he was able to identify biomarkers capable of predicting therapeutic responses. Alongside fellow co-founder and Chief Business Officer Christine Du, the pair have driven the startup to the point of attracting the attention of AstraZeneca, signing a multi-target research collaboration worth up to $555 million. The deal will apply Algen’s AI-driven CRISPR gene modulation platform to uncover novel therapeutic targets with an initial focus on immunology and chronic inflammatory disease, and the potential to expand into age-related conditions.
Both Levine and Doudna have worked alongside Carl June, with Doudna a co-author of June’s in a 2023 Cell study titled “Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells”, and Levine working in June’s labs since the 1990s. June continues to involve himself in cell therapy manufacturing too and has a place on the Board of Advisors at integrated development and manufacturing organization Cellares. I also spoke recently with Cellares CEO Fabian Gerlinghaus, who shared that June’s perspective is what reinforced his conviction to invest in automated cell and gene therapy manufacturing.
Gerlinghaus confirmed that a focus on the right technology is what led to some pretty impressive collaboration agreements, including with Bristol Myers Squibb and Kite/Gilead. “The $380 million global manufacturing deal with Bristol Myers Squibb was a watershed moment,” he said. “It showed that our technology and model were credible at commercial scale.”
Collaboration between therapy developers and tech specialists seems, so far, as a fairly reliable augur of success. Another recent collaborative effort sees Cleveland-based cell processing biotech Cell X Technologies integrate BioLamina’s defined recombinant laminin reagents (laminin-521 and laminin-511) into Cell X’s automated Celligent platform. With the aim of improving reproducibility and standardization in induced pluripotent stem cell (iPSC) culture and differentiation, this partnership seeks to reduce operator-dependent variability and support GMP-compliant workflows to enhance process consistency as regenerative therapies scale. Another example of how automation, imaging, and AI combine to continue to advance cell and gene therapies, Cell X CEO Lynn Frick said, “Our collaboration with BioLamina reflects a shared vision of reducing variability and increasing successes, especially during the critical transition from research to clinical application."
Such is the speed at which these technologies are developing, a stake in the CGT space appears not to be for everybody. The Board of Directors at Galapagos, for example, is actively exploring strategic options for its cell therapy division, including a possible sale. Several non-binding bids have already been received.
Elsewhere, a Form 8-K, filed by Rocket Pharmaceuticals with the US Securities and Exchange Commission, announces a voluntarily withdrawal of the company’s application to the FDA for approval of mozafancogene autotemcel (Fanskya or RP-L102). This therapy was being developed to treat Fanconi Anemia, a rare genetic disorder.
Rocket explained that the withdrawal is “based on business and strategic considerations and does not reflect concerns regarding the safety or efficacy profile” of the treatment. Rocket had already stopped funding new work on this therapy in mid-2025 and has pulled a similar application from European regulators. Though the data so far looks safe and promising, the company is preferring to focus resources on programs with better commercial and regulatory prospects.
Whether these two latter examples represent a failure to invest in the kind of partnerships technology that can keep pace with the therapies themselves, or other intrinsic bottlenecks, Adrienne Mendoza, Chief Operations Officer at BBG Biotherapies and Senior Vice President at BioBridge Global, says the role of automation in CGT manufacturing has too much potential to ignore: “Automation does show real promise in unlocking efficiencies, lowering costs, and eventually supporting distributed manufacturing models, but the real payoff will occur when we get all the way to fully closed automation, where every consumable is integrated and the process can move outside of heavy, resource-intensive cleanrooms. That’s where true scale – at the lowest possible cost, and most accessible to patients and donors – becomes possible.”
With its ongoing successes and failures, fascinations and innovations, watching the CGT industry develop is a bit like binge-watching your favorite TV box set. As we await the next crucial plot twist, I will leave you with more words of wisdom from Levine himself: “It’s about honoring the heroism of clinical trial patients, raising awareness of science, and combating misinformation. We have to move beyond scientific jargon, and learn to explain our work in everyday terms.”