Machine Learning and Single-Cell Technology Combined to Drive High-Performance Cell Line Development

OneCyte, which focuses on high-throughput single-cell analysis and cell line development technologies, and Kemp Proteins, which specializes in protein engineering and expression solutions, signed a strategic partnership agreement to deliver cell line development services for biopharmaceutical companies.

The collaboration brings together OneCyte’s proprietary single-cell platform for high-throughput and high-speed clone selection with Kemp Proteins’ molecular engineering capabilities, including its machine learning–driven platform, PROTiQ™.

Biopharma companies continue to face significant challenges in cell line development, including long development cycles, suboptimal yields, and high failure rates, particularly for novel and complex molecules, according to Konstantinos Tsioris, PhD, co-founder and president of OneCyte. These challenges can delay regulatory timelines and slow the progression of therapies into the clinic.

The OneCyte-Kemp partnership addresses these pain points by integrating predictive in silico design with rapid and high throughput experimental validation, say officials at both companies. As part of the workflow, amino acid sequences are evaluated using Kemp’s PROTiQ platform to assess developability risks, identify sequence liabilities, and generate structural insights.

The optimized candidates are then paired with OneCyte’s high-performance cell line development platform, which reportedly enables identification of elite clones with higher productivity.

Unlike traditional, rigid development workflows, this integrated approach is designed to adapt quickly to the evolving needs of new therapeutic modalities, notes Tsioris.

“By combining our single-cell technology with Kemp’s deep expertise in protein expression, we are confident that we can address the hardest challenges associated with new modalities, delivering faster timelines and industry-leading titers,” he continues.

“OneCyte’s class-leading single-cell technology, stacked on top of our molecular design and expression capabilities, will provide a powerful and differentiated solution for our global biopharma customers,” says Michael Keefe, CEO of Kemp Proteins.

The post Machine Learning and Single-Cell Technology Combined to Drive High-Performance Cell Line Development appeared first on GEN – Genetic Engineering and Biotechnology News.

STAT+: Up and down the ladder: The latest comings and goings

Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others. That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going.

And here is our regular feature in which we highlight a different person each week. This time around, we note that Proxygen hired Chiara Conti as chief scientific officer. Previously, she worked at Blueprint Medicines, where she was senior director.

But all work and no play can make for a dull chief scientific officer.

Continue to STAT+ to read the full story…

STAT+: Pharmalittle: We’re reading about Trump’s drug tariffs, a U.S.-U.K. pharma trade deal, and more

And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is rather modest so far. We plan to tidy up around the castle, promenade with the official mascots, and catch up on our reading. We also plan another listening party, where the rotation will likely include this, this, this, this and this. And what about you? The change of seasons opens up all sorts of possibilities, from long walks through woods to strolling along city streets to drives through the countryside. Of course, if the weather fails to cooperate, you could open a book, watch the telly, or spin a platter and dance about. Or maybe it is an opportunity to connect with someone special. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon. …

The Trump administration announced 100% tariffs on imported brand-name drugs — but with significant caveats, STAT explains. Many large drugmakers will not have to pay the tax because they struck deals with the U.S. to build manufacturing facilities here and lower the prices of their medications. Drugmakers that have not struck such deals but pledge to bring production to the U.S. can have tariffs reduced to 20% for the remainder of Trump’s term. The tariffs open a new front in the Trump administration’s efforts to rein in the pharmaceutical industry and in its push to bring manufacturing back to the U.S. The announcement comes as Trump has looked to emphasize his administration’s work to make prices — especially for medicines — more affordable ahead of the midterm elections.

Meanwhile, the Trump administration is negotiating more drug-pricing deals, now with smaller companies, according to STAT. The new talks offer a pathway for smaller pharmaceutical companies — those not included in the first round of deals — to pledge lower prices and potentially avoid tariffs or new pricing policies through Medicare. The negotiations suggest the administration is looking to replicate the strategy it used with larger drugmakers: extract voluntary, confidential agreements in pursuit of lower prices and more domestic manufacturing. They also offer smaller players in the sector the chance to cut a deal and gain more certainty about how they might be affected by federal policies. But the number of companies in talks with the administration remains unclear, as does whether or when the sides will reach agreement.

Continue to STAT+ to read the full story…

AI In Silico Multi-Omics Technique Cuts Therapeutic Development Costs

Bringing a drug from discovery through clinical trials takes too long and is too expensive, with preclinical costs alone estimated at $15 to $100 million. Employing artificial intelligence (AI) early in the process can lower those costs dramatically.

AI itself isn’t a panacea, though, Jayson Uffens, CTO and chairman of GATC Health, tells GEN. Instead, “Smart computing makes smart people smarter. There’s still a lot of expertise from people on the ground who bring a lot of value—maybe the ultimate value—to the mix.”

GATC Health, an AI-driven therapeutic discovery company, uses AI to raise the floor on opportunities to get high-potential compounds into human studies faster and thereby drive success.

Its proprietary approach to hit and lead identification and program derisking can cut preclinical development costs, according to Uffens, who maintains that the earlier AI is used in a program, the more dramatic the results.

The success GATC Health touts is based on deploying Operon™, the company’s proprietary AI platform. Operon deploys in silico models to simulate human biology and takes a multi-omics approach to analysis. That approach has allowed GATC to deliver three to five optimized compounds within six months, claims Uffens, versus the up to 48 months associated with traditional high-throughput screening methods.

Such acceleration occurs by using advanced in silico models to circumvent the “hundreds of thousands of dollars’ worth of experiments performed to get a hit and, ultimately, a lead,” Uffens says.

Rather than relying upon one huge model, he elaborates, “We attack the problem from multiple facets, looking at individual problems with various models and different architectures…and coordinate hundreds of AI models to answer different questions. That’s the starting point. There’s a lot of value in how we curate and parameterize our data in those specific contexts.”

The company also launched the Derisq™ AI Report, an in-depth analysis of drug candidates that highlights safety concerns, efficacy, and non-obvious risks early, while decision-makers can still modulate those risks.

This predictive intelligence layer is, in fact, a key element of GATC’s clinical trial insurance product. Underwritten by Medical and Commercial International (MCI) under the Lloyd’s of London framework, this insurance product leverages GATC’s predictive capabilities to identify risk. It reimburses the full cost of the trial if safety or efficacy endpoints aren’t met.

Typically, MCI’s preclinical trial insurance clients would provide that company with the relevant trial information, which would be run through the Derisq tool as part of their risk analysis.

Buyers for this insurance tend to be biopharma companies that aren’t large enough to self-insure their own trials. “Capital is expensive for them,” Uffens points out. “The insurance product is there to help them lower the cost of capital and open capital doors that may not be open otherwise.”

Multiomics to Discovery

What’s different about GATC’s approach to AI, Uffens says, is that “We come in, generally, as outsiders.” The founding team includes computer scientists as well as those with strong biology and genetics backgrounds, but not necessarily industry experience.

“We built our technology originally as a genetics interpretation platform,” he recalls, “and expanded it to find additional value.” The company was formed officially in 2020.

The turning point came when GATC became involved in a failed, big pharma program for addiction research.

“(The big pharma company) hadn’t found a solution, but had really valuable data and samples. A partner of ours was working with it to identify biomarkers and thought we could validate them. We discovered that not only could we validate the biomarkers, but we could also identify the therapeutic targets. That’s how we moved from multi-omics analysis into discovery,” Uffens recalls.

Moving forward, “We want to empower researchers,” he says. This means not only helping clients advance existing programs but also by identifying potentially more valuable targets.

Working with GATC

GATC’s key partners most likely will be biotech rather than big pharma, Uffens predicts. And, he notes, “We’re fairly agnostic to therapeutic area.”

“Most of our customers have called us because they want to realize the benefits of AI sooner rather than later,” Uffens says. “There is a lot of risk in the space. Folks who are willing to adopt AI at this stage…are looking for additional help before they risk more capital…” to solve particular challenges.

For a company to begin working with GATC, he explains, “The data we’re looking for is very similar to what they would include in an Investigational New Drug (IND) package. The earlier they are in the process, the less data they will have, but, at a minimum, we need some particulars on their therapeutic’s chemistry and the intended mechanism of action.”

Challenges

Drug development is a difficult space with plentiful challenges, he admits. Therefore, “We approach things as a tech company. We iterate through a problem and find where we can succeed or fail as quickly as we can to develop a solution. We’ve gone through multiple generations of architectures, finding ways that work best.”

The next milestone is to accumulate multiple successes with Operon and Derisq in human trials. “‘Wins in humans’ is our [next] frontier,” he says. That includes wins for its insurance underwriting partners as well as for companies working directly with GATC to advance therapeutics to human trials.

As part of that goal, GATC and BioAtla are closing a deal for a Phase III trial of ozuriftamab vedotin for oropharyngeal squamous cell carcinoma and to further develop conditionally active biologic senolytic therapies. Termed a special purpose vehicle transaction—a financial entity designed to hold specific assets that last for the life of the project—the $40 million deal formed Inversagen AI, LLC, to leverage the strengths of the founding companies.

“GATC and BioAtla are equal partners in Inversagen,” Uffens says. “GATC will own a percentage of ozuriftamab vedotin and a larger stake in future joint discoveries,” thus potentially discovering new therapeutic combinations that may be effective as conditionally active biologics.

Currently, the GATC is fine-tuning its own project prioritization. “The AI landscape is both beneficial and challenging,” Uffens acknowledges. “People have certain expectations about what AI can and should do, how it works, and how they might adopt it. Getting them to hear our unique perspective comes back to our focus on wins in humans.”

The post AI <i>In Silico</i> Multi-Omics Technique Cuts Therapeutic Development Costs appeared first on GEN – Genetic Engineering and Biotechnology News.

STAT+: Small drugmakers, facing threat of tariffs, negotiate pricing deals with White House

WASHINGTON — The Trump administration is negotiating new drug-pricing deals, now with smaller companies, according to three people with knowledge of the meetings, including a White House official.

The new talks offer a pathway for smaller pharmaceutical companies — those not included in the first round of deals — to pledge lower prices and potentially avoid tariffs or new pricing policies through Medicare.

The new negotiations suggest the administration is looking to replicate the strategy it used with larger drugmakers: extract voluntary, confidential agreements in pursuit of lower prices and more domestic manufacturing. They also offer smaller players in the sector, which have faced substantial uncertainty about how federal policies would affect them, the chance to cut a deal and gain more certainty about how they might be affected by federal policies. 

Continue to STAT+ to read the full story…

STAT+: Trump announces 100% tariffs on brand-name drugs, with plenty of carveouts

WASHINGTON — The Trump administration announced Thursday 100% tariffs on imported brand-name drugs — but with significant caveats.

Many large drugmakers won’t have to pay the tax because they’ve struck deals with the U.S. to build manufacturing facilities here and lower the prices of their medications. Drugmakers that haven’t struck those deals but pledge to bring production to the U.S. can have their tariffs reduced to 20% for the remainder of Trump’s term. 

The tariffs open a new front in the Trump administration’s efforts to rein in the pharmaceutical industry and in its push to bring manufacturing back to the U.S. The announcement comes as Trump has looked to emphasize his administration’s work to make prices — especially medicines — more affordable ahead of the midterms.

Continue to STAT+ to read the full story…