Bits In Bio Weekly - December 3rd 204
Updates From and Around the Bits in Bio Community
📢 Highlights
Harvard's TITAN Pushes Computational Pathology With New Foundation Model
Novartis’ $1B Bet on PTC518 as Innovative Huntington’s Disease Treatment
Instadeep Unveils Billion-Parameter Nucleotide Transformers for Multi-Species Prediction
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TITAN - a Multimodal Whole Slide Foundation Model for Pathology
Researchers from Harvard University introduced TITAN, a multimodal whole-slide image (WSI) foundation model designed to address challenges in computational pathology. WSIs are digitized, high-resolution scans of pathology slides, offering a comprehensive view of tissue samples at a microscopic level. TITAN leverages self-supervised learning and vision-language alignment to encode WSIs into versatile feature representations, enabling applications such as rare disease retrieval and cancer prognosis without requiring extensive clinical labels. The model is trained on a large dataset of WSIs and synthetic captions, demonstrating superior performance across various clinical tasks compared to existing models. The code is available on Github.
Behind the Deal: Novartis’ $1B bet on PTC518 to tackle Huntington’s disease
The Swiss pharma giant Novartis has acquired global rights to PTC Therapeutics’ PTC518, an oral therapy for Huntington’s disease (HD), for $1 billion upfront and up to $1.9 billion in milestones. This move shows Novartis’ commitment to addressing HD, a fatal disorder with no disease-modifying treatments. PTC518 targets mutant huntingtin protein, the root cause of HD, offering hope for a transformative therapy. Interim Phase 2 PIVOT-HD data from June showed dose-dependent reductions in huntingtin protein and encouraging trends in clinical outcomes. With the FDA’s Fast Track designation, PTC518 benefits from an expedited development timeline, improving the potential for quicker returns. The therapy’s oral formulation and strong CNS penetration address safety concerns that have hampered prior candidates. For Novartis, this deal builds on its neuroscience expertise and strengthens its position in HD innovation. As one of the most advanced HD splicing modulators, PTC518 contrasts with approaches like uniQure’s gene therapy AMT-130, which required slow trial progression due to initial safety concerns. By targeting a market with high unmet need and significant commercial potential, Novartis is making a calculated bet with the potential for transformative impact and high reward.
Instadeep’s Nucleotide Transformer Advances Genomic AI
UK-based Instadeep has unveiled Nucleotide Transformer in a recent Nature article, a set of foundation models for encoding genomic sequences. These models, ranging from 500 million to 2.5 billion parameters, were trained on three comprehensive datasets: the human reference genome, 3,202 diverse human genomes, and 850 genomes spanning multiple species. This training strategy “enables the models to identify functionally significant genomic patterns across species, enhancing their performance on human genome prediction tasks.” The team has also demonstrated that these models can be efficiently fine-tuned for specific applications, such as predicting molecular phenotypes from DNA sequences. The pre-trained models and inference code are available on Github. Models and example notebooks are also available on HuggingFace.
Series D of $115M Propels Maze's APOL1 Therapy Development
San Francisco based Maze Therapeutics has raised $115 million in Series D funding, led by ARCH Venture Partners and Third Rock Ventures, to accelerate development of MZE-001, its lead candidate for APOL1-mediated kidney diseases. APOL1 variants are linked to up to 70% of chronic kidney disease cases in African-American populations, presenting a significant unmet medical need. Maze’s approach integrates genomic insights with small molecule design, aiming to address conditions like focal segmental glomerulosclerosis (FSGS). The funding will also advance other pipeline programs targeting genetically defined diseases.
ARPA-H Backs Harvard's Innovative Immune Organ Cancer Therapy
The Wyss Institute at Harvard has won an elusive ARPA-H grant to advance INODES, a project focused on creating implantable immune organs for personalized ovarian cancer therapy. INODES integrates biomaterials and engineered immune cells to amplify the body's ability to recognize and attack tumor neoantigens. Led by Dr. Goyal and supported by researchers including Dr. Sudip Paudel and Abdul Isaacs, the project targets a cancer type with a <50% 5-year survival rate, addressing an urgent need for effective treatments. ARPA-H funding emphasizes the federal support for the potential of engineered immunotherapies to redefine cancer care through innovative solutions.
Strategic $500M Agreement Transfers Relay's RLY-1971 to Elevar
Relay Therapeutics has partnered with Elevar Therapeutics to transfer rights to RLY-1971, a SHP2 inhibitor targeting rare cancers, in a deal valued at up to $500 million. Preclinical studies revealed RLY-1971's potential to inhibit tumor growth driven by oncogenic pathways, positioning it as a promising candidate for hard-to-treat cancers. Elevar will manage late-stage clinical development and global commercialization, while Relay reallocates resources to other assets in its AI-enabled pipeline. This transaction aligns with Relay’s strategy to streamline its portfolio while expediting the drug's market entry, which is aligned to the firm's origin with David E Shaw and his eponymous multibillion dollar finance firm.
$200M Keros Acquisition Strengthens Takeda's Blood Disease Pipeline
Takeda has paid $200 million upfront to acquire global rights to KER-050, Keros Therapeutics’ promising hematology asset. KER-050 targets the TGF-beta signaling pathway to address ineffective red blood cell production in myelodysplastic syndromes and beta-thalassemia, conditions affecting over 100,000 patients annually worldwide. With potential milestone payments surpassing $1 billion, the deal positions Takeda to challenge Bristol Myers Squibb’s Reblozyl in a hematology market projected to grow 7.5% annually to $5.7 billion by 2028.
🗓️ Upcoming BiB Events
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Minneapolis-St Paul Get Together - Tuesday, December 3, 5:00 PM at Pryes Brewing Company
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BiB x TBT: San Diego Mixer - Wednesday, December 4, 6:30 PM at Fall Brewing Company
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Bits in Bio - Austin Texas Meetup - Thursday, December 5, 6:00 PM at Meanwhile Brewing
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Building hardware for Biology - Monday, December 9, 7:00 PM at BioLabs Hôtel-Dieu Paris
Thank you Gustave Ronteix, Christophe Tallec, and Estelle Botbol for organizing!
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