![A view of some of the exhibitors in attendance at BIO 2025 [Uduak Thomas]](https://www.genengnews.com/wp-content/uploads/2025/06/IMG_BIO-2025.jpg)
A view of some of the exhibitors in attendance at BIO 2025 [Uduak Thomas]
At this year’s BIO International Convention, held June 16–19 in Boston, MA, two companies, 64x Bio and Fauna Bio, introduced new products aimed at improving therapy delivery and identifying novel drug targets for a range of human diseases, respectively.
For its part, 64x Bio launched AAV Apex suite, a product designed to address bottlenecks in gene therapy manufacturing. Through the suite, the company provides suspension-adapted HEK293 cell lines for transient transfection alongside optimized production processes that deliver AAV titers exceeding E15 vg/L. This performance has been independently confirmed by partners across various serotypes and therapeutic payloads, according to the company.
Meanwhile, Fauna Bio launched Fauna Brain, an artificial intelligence platform that is helping the company speed up efforts to identify novel targets for drug development. According to the company, Fauna Brain is a multi-agent AI system capable of autonomously executing complex research tasks that typically require experts. It combines Fauna’s internal datasets with public databases and scientific literature to identify and score drug targets. The company claims that the tool can score a single target in a little over two minutes, and that it can screen multiple targets in parallel at a fraction of traditional R&D costs.
Targeting AAV manufacturing’s pain points
The seeds for what would become 64x Bio’s technology were first planted during CEO and co-founder Alexis Rovner’s postdoctoral research in the Harvard University laboratory of George Church, PhD. Church is listed as a co-founder of 64x Bio as well as a member of its scientific advisory board. The company was founded in 2017 and has raised over $59 million in seed funding.
At the time, conversations with drug developers made clear just how much of a challenge manufacturing advanced therapies really was, Rovner, PhD, told GEN in an interview at the BIO meeting. “There were just gigantic bottlenecks in manufacturing and scalability,” she said.
The underlying technology for the AAV Apex Suite is the company’s VectorSelect platform, which houses a large data map of cellular programs that are responsible for boosting yield. Specifically, it contains insights into the genetic and metabolic mechanisms that inform cell line engineering and nutrient formulations for media components that maximize cell line productivity.
Rather than evaluating and tweaking individual genes in a multi-well format as some had tried to do, “we invented a way to do pooled screens for AAV productivity” using barcoding technology, Rovner explained. That made it possible to assess “many different perturbations at a time in a single experiment” and create a map of which “genetic and metabolic perturbations are really important and the right combinations for maximizing AAV production.”
To date, 64x Bio has signed strategic collaborations with five global biopharma and contract development and manufacturing organizations who are interested in the AAV Apex suite. The list includes Charles River Laboratories, which tapped the company as a partner for their incubator program, meaning it can offer 64x Bio cell lines to other participants in the program.
64x Bio is also interested in partnering on custom projects. The company has shared data publicly on its website showing that its technology does well at creating “cell lines that are one-size fits all and work for a number of different serotypes and therapeutic payloads,” Rovner said. However, “there are a lot of novel capsids that are being engineered now … and very specific niche cases that maybe our cell lines today won’t work for.” Maybe in the future they will, but in the meantime, we can partner to create a cell line that works.”
Other plans include expanding the Apex suite beyond transient production, which uses plasmids, to include stable cell lines that do not require plasmid transfection. In both cell line types, they will use computational tools to identify synergistic combinations of metabolic and genetic perturbations that further boost production yields. Additionally, they plan to support other modalities beyond AAVs, such as lentiviruses.
Early feedback from partners who have used the AAV Apex suite platform has been positive. For Rovner, it underscores the importance of developing tools that address manufacturing bottlenecks at a cost-effective price point. “We have gene therapies that have a lot of potential, but we can’t scale these medicines … because it’s just very hard to produce in general [and] also the cost to do so is enormous,” a fact that “constrains indication selection for a lot of companies,” she said. “If we don’t solve it, we’re not going to be able to see the true promise of these medicines. Our goal is to really enable our partners to bring down COGS to a level where they can choose broader indications, which ultimately allows for these medicines to reach more patients.
From animal biology to novel human treatments
Before earning her doctorate in cancer biology from Stanford University, Ashley Zehnder, PhD, worked as a veterinarian for exotic animals. “I got all my clinical training in nontraditional species,” she told GEN in an interview. Over time, she became interested in how “different diseases present across different species” as well as in the “molecular mechanisms at play within those differences.” That interest led her to a post-doctorate at Stanford focused on the intersection of animal health and human diseases.
That’s where Zehnder, who is now the CEO and co-founder of Fauna Bio, which launched in 2018, met two of the company’s co-founders. One of them, Linda Goodman, PhD, who also now serves as Fauna Bio’s CTO, was interested in studying how genes evolve over time. The other, Katie Grabek, PhD, who now serves as the company’s CSO, was studying genes involved in hibernation cycles in mammals. While working together, they realized that insights from animal biology could pinpoint “really unique ways to resist and repair, even reverse disease that could be really useful to improve human health.”
Genomic datasets from both animals and humans are incredibly large and complex, and the partners quickly realized that they needed powerful computational tools to effectively mine and compare data across species. This led to the development of Convergence, the underlying AI platform that powers Fauna Brain, the company’s latest product. Convergence uses a series of applications to integrate data from sources like the Zoonomia Consortium, the UK Biobank, and PubMed. Currently, it combines transcriptomic, proteomic, and epigenomic data from 292 species and 24 tissues collected at 21 time points.
Relying on comparative genomic insights gleaned from mammals with extreme phenotypes, such as hibernators, regenerators, and species resistant to fibrosis, cancer, or metabolic dysfunction, the company’s AI identifies potentially druggable targets in humans.
Convergence powers Fauna Brain, the company’s multi-agent AI system, which is only used internally. Essentially, the agents are trained to use Convergence much like the company’s own expert drug discovery team would, but much faster. “Our team still checks the work, but it takes a lot of the manual work that they would do to use the platform,” Zehnder said.
Incorporating AI agents allows Zehnder and her team to work on a much broader set of discovery projects and take on more partnerships. “Typically, we’re working with big pharma to address an area of unmet need where we have really unique data resources,” she explained. The company is currently working on projects in cardiovascular disease and obesity with pharma partners, including Eli Lilly.
“We work with pharma to figure out what are the right species [and] what tissue time points we are going to look at. Then we do the discovery using our own platform and we present target concepts to pharma.” The company also has in-house wet lab capabilities that let them perform validation experiments.
Fauna Bio is currently developing an internal pipeline of drugs that includes a program in neuroprotection. “What’s really interesting is that during hibernation, we see an increase in tau phosphorylation.” Essentially, there were changes in the tau protein that are similar to early stages of Alzheimer’s in people. “In hibernating mammals, this is a change that’s reversed about every two weeks” and results in recovered neuronal connections in the brain, she explained. They have observed this neuroplasticity in many species that hibernate, including lemurs and other primates. ”We’re tapping into these core protective mechanisms through this lens of hibernation.”
Other internal programs focus on retinal disease, and there are potential applications in space health and radiation resistance. Currently, the company has a lead candidate, Faun1083, derived from genes linked to hibernation physiology, that has shown improvement in diastolic dysfunction in a preclinical model of heart failure with preserved ejection fraction. Other programs explore neuron survival and metabolic reprogramming under extreme conditions.
“We’re pretty open in terms of [delivery] modality,” Zehnder said. “We consider ourselves modality agnostic because we’re really looking for the strongest biology.”
