10 Cutting-Edge Startups Focusing On RNA Research
ALSO: Weekly tech+bio news; Google's Novel TPUs Power RNA-focused AI Foundation Model; NVIDIA’s AI Platform Grows; Google Introduces Med-Gemini
Hi! I am Andrii Buvailo, and this is my weekly newsletter, ‘Where Tech Meets Bio,’ where I talk about technologies, breakthroughs, and great companies moving the biopharma industry forward.
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Now, let’s get to this week’s topics!
Weekly tech+bio highlights
💰 Johnson & Johnson acquires Proteologix for $850M, gaining access to bispecific antibody programs, including Phase I-ready PX128 for atopic dermatitis and asthma.
The acquisition enhances J&J's immunology pipeline with PX128 and preclinical PX130, targeting IL-13 and cytokines for moderate to severe atopic dermatitis and asthma.
⚡ Recursion Pharmaceuticals launches BioHive-2, the fastest supercomputer owned by a pharmaceutical company, developed with Nvidia. Ranked No. 35 worldwide, this supercomputer enhances AI-driven drug discovery, allowing Recursion to train larger AI models more efficiently and support multiple parallel AI projects.
🔬 AstraZeneca adds a novel heart failure target to its discovery portfolio, discovered using BenevolentAI’s AI-drug discovery platform and validated by AstraZeneca, marking significant progress in the field of target identification.
💰 Nabla Bio closes a $26M Series A round led by Radical Ventures and announces over $550M in collaborations with AstraZeneca, Bristol Myers Squibb, and Takeda to develop protein drugs.
🔬 Erasca cuts 18% of its workforce and halts three clinical programs, focusing on KRAS inhibitor and molecular glue therapy, licensing them for $22.5M upfront.
🔬 Sartorius and NVIDIA expand their collaboration to develop predictive AI models for stem cell-derived organoids, aiming to replace animal models in drug discovery and precision medicine, leveraging NVIDIA’s Clara suite and DGX platform for advanced biopharmaceutical manufacturing and therapeutic development.
✅ Karius Inc. secured $100M in funding and announced its rapid Karius Test received FDA Breakthrough Device designation, expediting review and development for diagnosing pneumonia and lung infections in immunocompromised patients.
🔬 Insilico Medicine, in collaboration with Fosun Pharma, initiated the first-in-patient dose of ISM8207, a novel QPCTL inhibitor which targets a protein involved in immune evasion, in a Phase I clinical trial for advanced malignant tumors.
⚠️ MacroGenics reports significant safety concerns in Phase II trial of vobramitamab duocarmazine for metastatic prostate cancer, with over 50% experiencing serious adverse events and five deaths, leading to a 70% stock drop despite some efficacy signals.
💰 Accenture invests in Turbine to enhance AI-powered cell simulations for biopharma research, aiming to accelerate drug R&D through Turbine's Simulated Cell™ platform. Terms of the investment remain undisclosed, with Turbine joining Accenture Ventures’ Project Spotlight to leverage Accenture’s domain expertise and enterprise client access.
🔬 Galapagos partners with Blood Centers of America to enhance GMP-compliant, decentralized CAR-T therapy production, reducing vein-to-vein time to seven days for its haemato-oncology program.
📉 Ionis and Biogen discontinue their experimental ALS drug BIIB105 after it failed to show clinical benefits in the Phase I/II ALSpire trial, despite reducing ATXN2 protein levels.
Now Google's Novel TPUs Power RNA-focused AI Foundation Model
During Google I/O 2024, Canadian "techbio" company Deep Genomics announced that it is using Google Cloud’s next generation Tensor Processing Units (TPUs) in the development of its AI foundation model.
Deep Genomics is the creator of BigRNA, the first AI Foundation model for decoding RNA biology and designing therapeutics (as the company claims).
I will soon be publishing my interview with Dr. Brendan Frey, Founder and Chief Innovation Officer at Deep Genomics.
Brendan is an incredibly interesting guy who, among a litany of major contributions, was an early pioneer in the development of ML & AI to accurately determine the consequences of genetic mutations.
Stay tuned!
NVIDIA’s AI Platform Grows
NVIDIA expands its BioNeMo platform with new foundation models and microservices, enhancing AI-powered drug discovery by supporting over 100 firms with tools for DNA sequence analysis, protein structure prediction, and RNA data interpretation.
The models, including DNABERT, scBERT, and EquiDock, are available as microservices through NVIDIA NIM, facilitating easy integration into existing drug discovery workflows across various cloud platforms.
Introducing Med-Gemini
Google Health introduced Med-Gemini, a new family of AI research models tailored for medicine, achieving 91.1% accuracy on the MedQA benchmark and generating reports for 2D and 3D medical datasets, including 3D brain CTs. These models excel in analyzing medical images, videos, genomics, and health records, with advanced reasoning and long-context abilities for summarizing health records and research papers, and predicting health outcomes from genomic data using polygenic risk scores.
10 Cutting-Edge Startups Focusing On RNA Research
For many years, the focus of therapeutic developments and scientific research was directed at the genome and proteins. Yet, there is a completely different level of cell regulation: the transcriptome level, presented by ribonucleic acids (RNAs).
RNA molecules are not just intermediates between DNA and proteins, in fact, they also carry out enzymatic functions, control gene expression, and perform other functions in cells.
Major breakthroughs in transcriptomics have been made since the beginning of the century: the discovery of RNA interference blew up the industry and the Nobel Prize in Physiology or Medicine 2006 was awarded jointly to Andrew Z. Fire and Craig C. Mello "for their discovery of RNA interference - gene silencing by double-stranded RNA."
RNA interference is a gene silencing mechanism promoted by small siRNA molecules that are also exploited in patisiran and inotersen – first-in-class RNA therapeutics. Both drugs target transthyretin, a protein involved in the pathogenesis of transthyretin-mediated amyloidosis, and reduce the expression of its mutant version.
Today, efforts by companies in the transcriptomics space are typically directed in one of the following ways:
RNAs that modulate DNA,
RNAs that target proteins,
RNAs that encode proteins,
modalities, like small molecules, that modulate RNA or related transciption machinery (e.g. splicing)
delivery of RNA-based therapies
other strategies, like non-coding RNAs
Among existing modalities – ASOs, single-stranded antisense oligonucleotides that block mRNA translation, siRNAs and microRNAs that degrade protein-encoding mRNAs, RNA aptamers that tightly bind specific spots on proteins and regulate their functioning, small molecules targeting RNAs or transcriptome-related proteins, and so on.
In addition to that, last year showed a real boom in mRNA vaccines, with coronavirus vaccines and cancer vaccines being the most prominent representatives.
While there are numerous companies developing RNA-based therapeutics and vaccines, including major corporations like Pfizer, BioNtech, Moderna, etc., below I am listing several biotech startups to keep an eye on.
Some of them are platform companies powered by artificial intelligence (AI):