Bits In Bio Weekly - June 3rd 2025
Updates From and Around the Bits in Bio Community
📢 Highlights
FDA deploys secure AI assistant, Elsa, to tackle staffing challenges and accelerate scientific reviews
Sanofi doubles down on rare disease strategy with $9.5B Blueprint deal targeting AI-driven drug development
Stanford and Genentech unveil Biomni, an open-source AI agent achieving human-level biomedical research
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FDA Launches AI Tool ELSA to Accelerate Scientific Reviews
The FDA has launched ELSA, a generative AI tool designed to expedite internal scientific reviews and regulatory tasks. Introduced ahead of schedule and under budget, ELSA assists with summarizing adverse events, comparing drug labels, and generating database code, significantly reducing task durations from days to minutes. Operating within a secure GovCloud environment, ELSA ensures that sensitive data remains confidential and is not used for external model training. This initiative is part of the FDA’s broader strategy to integrate AI across all its centers by June 30, aiming to enhance efficiency amid staffing challenges. The rollout is led by Chief AI Officer Jeremy Walsh and Sridhar Mantha, focusing on expanding ELSA’s capabilities based on user feedback.
Behind the Deal: Sanofi’s $9.5B Blueprint Buyout Signals Rare Disease and AI Push
Sanofi’s $9.5 billion acquisition of Massachusetts-based Blueprint Medicines marks a defining moment in the rare disease and hematology space. The deal centers on Ayvakit, Blueprint’s precision therapy for advanced systemic mastocytosis (SM), which has shown strong commercial traction since its 2021 FDA approval. This acquisition allows Sanofi to deepen its rare disease portfolio with a high-margin, high-growth product that complements its existing specialty care business.
The timing is critical: Ayvakit sales are accelerating, driven by label expansion and increasing diagnostic awareness, just as Sanofi seeks growth engines beyond immunology and vaccines. Analysts estimate peak sales could exceed $2 billion annually, making this a revenue generating asset with de-risked clinical fundamentals. Blueprint also brings a proprietary discovery engine that uses computational chemistry and structure-based design to engineer kinase inhibitors targeting genomically defined diseases. This fits into Sanofi’s broader AI strategy, as it continues to build digital capabilities through ventures like its partnership with Owkin and the BioDrive AI platform. Beyond product synergy, this move positions Sanofi ahead of rivals in precision hematology and underscores the increasing role of AI in de-risking and accelerating drug development. This isn’t just a pipeline play—it’s a revenue and platform bet rolled into one.
Biomni: A General-Purpose Biomedical AI Agent
Developed by researchers at Stanford’s Computer Science and School of Medicine departments, and Genentech, Biomni is a general-purpose biomedical AI agent designed to perform complex research workflows autonomously. This includes anything from hypothesis generation and protocol design to multi-omics and wearable data analysis. Biomni operates within a unified action environment (Biomni-E1) of 150 tools, 105 software packages, and 59 databases, curated from over 2,500 bioRxiv papers. Benchmarked against expert baselines, it achieved human-level performance on multiple biomedical QA tasks. It demonstrated strong generalization across eight real-world tasks, including genome-wide association studies (GWAS), drug repurposing, and the diagnosis of rare diseases. The system is (almost) fully open-source and will be available for academic use via Github.
Strong Phase 1 safety results for ENV-294 by Enveda, moving to atopic dermatitis efficacy trial
Enveda has achieved a significant milestone by successfully completing Phase 1 safety trials for ENV-294, marking the first drug candidate derived from AI-discovered natural chemistry to reach clinical trials. The first-in-class oral anti-inflammatory demonstrated favorable safety and tolerability with no dose-limiting toxicities or serious adverse events across all dose levels in healthy volunteers. ENV-294 aims to combine "JAK-inhibitor-like efficacy with IL4/IL13-like safety," potentially addressing the significant unmet need for safe oral therapies in atopic dermatitis—a condition affecting over 200 million people worldwide. The compound showed dose-proportional pharmacokinetics supporting once-daily dosing, with the Safety Review Committee endorsing advancement to Phase 1b trials in patients with moderate to severe atopic dermatitis, expected to begin in the second half of 2025. Enveda's platform leverages a searchable library of over 1.5 million natural compounds, representing a novel approach to drug discovery that could potentially bypass some of the safety concerns plaguing current oral inflammatory treatments.
Canadian biotech prepares to advance pioneering AI-designed peptide therapeutic into clinical trials
Toronto-based ProteinQure has secured an $11 million Series A round led by Heron Rock Fund to advance what the company claims could be the first AI-designed peptide therapeutic into human trials. The funding will support a multicenter Phase 1 trial for PQ203, a peptide-drug conjugate targeting triple-negative breast cancer through the sortilin receptor, with enrollment of 70-100 patients planned across prestigious cancer centers including Princess Margaret, MD Anderson, and Yale. The company's ProteinStudio™ platform integrates machine learning, structural biology, and atomic-level simulations to design therapeutic peptides using thousands of non-natural amino acids— addressing the traditional limitations of peptide drugs through computational approaches. PQ203 specifically targets tumors resistant to current antibody-drug conjugates like Trodelvy, with robust preclinical data in patient-derived xenograft models demonstrating superior efficacy compared to standard treatments. Lead investor Tom Williams of Heron Rock Fund described the milestone as witnessing "the birth of a new drug class," noting ProteinQure's remarkable capital efficiency in advancing from concept to clinic with total funding of just $16 million across seed and Series A rounds.
Brain-computer interface leader closes huge funding round as 5 paralyzed patients successfully use implants
Elon Musk's BCI company, Neuralink, has closed a $650 million Series E funding round led by heavyweight investors including Sequoia Capital, ARK Invest, and Founders Fund, bringing the company's valuation to approximately $9 billion. The funding comes as five patients with severe paralysis are actively using Neuralink's "Link" implant to control computers and communicate through thought alone— with one patient reportedly playing Counter-Strike using only neural signals. Since its 2023 Series D round, the company has launched global clinical trials across three countries and received FDA breakthrough device designation for its speech restoration capabilities. The timing couldn't be more competitive, as Texa-based rival Paradromics simultaneously announced its first human implant test, highlighting the intensifying race to commercialize brain-computer interfaces. While Neuralink's 1,024-electrode system aims to restore independence for patients with paralysis, the company faces ongoing regulatory scrutiny and internal challenges despite its high-profile progress demonstrations.
23&me's ex-CEO contests bankruptcy sale process with undisclosed Fortune 500 partner
Just when you thought all was said and done, Anne Wojcicki, cofounder and estranged CEO of South San Francisco-based 23andMe, has demanded a federal bankruptcy court to reopen the company's asset auction, claiming she has financial backing from an unnamed Fortune 500 company with over $400 billion in market capitalization and $17 billion in cash. The challenge comes after Regeneron Pharmaceuticals won the bankruptcy auction with a $256 million bid, beating Wojcicki's shell non profit ‘TTAM Research Institute’ bid of $146 million—though Wojcicki has made baseless claims her consortium was unfairly capped at $250 million and prepared to bid over $280 million. The genetic testing company filed for Chapter 11 bankruptcy in March following declining consumer demand and a devastating 2023 data breach that exposed sensitive genetic information of millions of customers, causing share prices to plummet from a $3.5 billion SPAC valuation in 2021 to around 88 cents. Wojcicki argues that 23andMe's advisors prematurely concluded the auction process due to "misplaced concerns" about TTAM's financial capabilities, while the company's debtors maintain the process was conducted fairly under independent director oversight. The court has set June 12 as the deadline for final proposals from both parties, with a proposed $10 million breakup fee for Regeneron if Wojcicki's bid ultimately prevails.
Brainreader Raises €6.6M to Expand Its AI-Powered MRI Platform for Alzheimer’s and TBI
Denmark’s Brainreader secured €6.6 million (~$7.3M) to accelerate the global rollout of Neuroreader, its AI-driven software for brain MRI analysis. Neuroreader, already CE-marked in Europe and FDA-cleared in the US, automatically measures brain volumes on MRI scans to help doctors catch subtle signs of neurodegenerative diseases like Alzheimer’s, as well as monitor traumatic brain injury progression. The new funding, led by Dahlgren Capital, will be used to scale commercial operations (especially in U.S. hospitals) and develop new clinical features as Brainreader integrates into routine radiology workflows The company is benefiting from tailwinds in both reimbursement and regulation: in the U.S., new insurance billing codes now cover AI-based brain volumetrics, and in Europe, updated medical device rules are encouraging adoption of AI imaging tools. With this capital, Brainreader aims to cement its position as a category-defining player in AI-enabled neurodiagnostics, bringing faster, more precise brain scan insights to clinics worldwide.
BioAge Labs finishes IND-enabling studies for BGE-102, targeting mid-2025 clinical entry
Emeryville-based BioAge Labs has completed IND-enabling studies for BGE-102, a brain-penetrant NLRP3 inhibitor that could potentially reshape obesity treatment. The orally available small molecule demonstrated impressive preclinical results, achieving roughly 15% weight loss as monotherapy—comparable to semaglutide—and over 20% when combined with GLP-1 receptor agonists in diet-induced obese mice. What makes BGE-102 particularly intriguing is its novel binding site and ability to achieve greater than 90% NLRP3 inhibition in the brain over 24 hours with a predicted human dose below 50mg once daily. The company aims to submit an IND application in mid-2025, with initial Phase 1 single ascending dose data anticipated by year-end. BioAge identified NLRP3 as a target through analysis of human aging cohorts, revealing that reduced NLRP3 activity correlates with greater longevity—a compelling example of how aging biology insights could translate into metabolic therapies.
TreeFrog Therapeutics Secures €30M from EU Investment Bank to Adv. Parkinson’s Cell Therapy
French biotech TreeFrog Therapeutics landed a €30 million financing from the European Investment Bank to turbocharge its cell therapy pipeline – chiefly a stem cell–derived treatment for Parkinson’s disease that’s aiming for first-in-human trials in 2027. The deal is structured as a mix of equity-like funding and venture debt (in three €10M tranches) under the EU’s InvestEU program, giving TreeFrog runway to push its lead Parkinson’s therapy into the clinic and expand its platform to other diseases. TreeFrog’s value proposition lies in its C-Stem technology, which uses microfluidics and 3D microtissues to mass-produce high-quality induced pluripotent stem cell (iPSC) derivatives – effectively solving a major bottleneck in cell therapy. The company’s approach (encapsulating cells in tiny alginate beads to mimic natural growth) promises safer, scalable production of transplantable cells, which could make regenerative treatments for conditions like heart failure or diabetes more feasible long-term. European officials tout this investment as a strategic boost for homegrown biotech innovation, signaling confidence that platforms like TreeFrog’s could “revolutionize healthcare” by curing diseases rather than just managing symptoms.
Insilico Medicine Announces Fifth AI-Generated Small Molecule Targeting ENPP1
Insilico Medicine has nominated its fifth AI-designed small molecule since 2024, targeting ENPP1, an enzyme known to suppress anti-tumor immunity. In a recent Nature Communications paper, the company presented an orally available ENPP1 inhibitor designed to treat solid tumors. By inhibiting ENPP1, the compound enhances anti-tumor immunity without the systemic toxicity typically associated with direct immune activation.Target identification was performed using PandaOmics, Insilico’s AI platform that integrates biological networks and text-based data to prioritize pathogenic targets. Using TCGA datasets, the team identified several tumor types where ENPP1 inhibition may be therapeutically beneficial.
To design the inhibitor, the company used Chemistry42, its generative AI drug design platform. Starting from a known ENPP1 inhibitor, the platform generated and optimized multiple candidate structures for binding affinity and drug-like properties through iterative refinement.In preclinical studies, the lead compound, ISM5939, demonstrated enhanced efficacy when combined with existing cancer therapies, including immunotherapy. This marks the fifth AI-generated preclinical candidate by Insilico Medicine since 2024. All nominated compounds have progressed to the IND-enabling stage. The company highlights that the AI-assisted design process reduced candidate nomination time to 12–18 months, compared to the traditional 2.5–4 years.
Vivodyne Raises $40M to Scale AI + Organoid Platform Aiming to Cut Drug Trial Failures
A Philadelphia/SF startup called Vivodyne has snagged $40 million in Series A funding led by Khosla Ventures to grow its robotic “human tissue” testing platform as an alternative to animal experiments. The company’s tech can cultivate thousands of tiny, lab-grown human organoids (think mini-organs on chips) and then use automated imaging and AI to test drug candidates on them in parallel. The big idea: replace unreliable animal models with human-relevant data early on, closing the “translational gap” behind the ~95% clinical trial failure rate for new drugs. With the new cash, Vivodyne is opening a 23,000 sq ft fully robotic lab in South San Francisco to meet surging demand from pharma clients and to capitalize on recent FDA/NIH support for non-animal testing methods. Notably, Vivodyne says most top 10 pharma companies already use its human tissue data in their R&D, hinting that industry appetite for organoid-based drug screening is real – and about to get a major capacity boost.
Automating Compounding at Pharmacies with “Pharma Printer” - CurifyLabs Bags €6.7M
CurifyLabs, a Finnish healthtech startup, raised €6.7 million to scale up its robotic drug compounding platform that can print personalized medications on-demand. Led by the Springvest investment group (plus a €1M innovation loan from Business Finland), the round will fund expansion to high-volume pharmacy partners – including plans to enter the U.S. – and commercialization of the company’s desktop Pharma Printer device. CurifyLabs’ system replaces the age-old practice of pharmacists manually mixing custom drug doses (for kids, cancer patients, people with rare needs) with an automated combination of pre-formulated bases, active ingredients, and a precision robotic dispenser. This automation reportedly makes compounding 4x faster and enables “limitless” dose and formulation flexibility, addressing the fact that standard pharma dosing often doesn’t fit many patients (half of medicines lack child-friendly versions, for example). With the global compounding market at ~€15B and growing, CurifyLabs is positioning its tech at the intersection of personalized medicine and pharmacy operations – aiming to bring bespoke drug manufacturing to scale without sacrificing quality or safety.
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April Journal Club – Cell and Gene Therapies, Wednesday, June 4th
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📰 Top Community Conversations
Bits in Bio community resource: RNA therapeutics deals Notion Page
In recent months there have been quite a few deals involving RNA drugs, with about $6B biobucks signed in just the past month. Elli Lilly has gone on quite a partnership spree right after opening a new $700m office for RNA therapeutics in seaport. Ionis and Anlylam have also churned along sending drug after drug into phase 3. We thus started to collect some databases of the moves in that field, here is the first list collecting the recent deals announced between biotech and pharma for RNA medicine, please ping Felix if some are missing!
🏢 New Job Openings
Postdoctoral Fellow, Machine Learning at the SickKids
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Lead Machine Learning Scientist and Fractional Chief Data Officer at Immuto Scientific
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