AI-powered drug discovery: update (III)
AI-powered drug discovery: update (III)
MetaphysicalCells: A newsletter about Science, Technology and AI Drug Discovery
This article is part of a series of articles dedicated to AI-drug discovery progress of drug candidates in preclinical and clinical phase. For the previous newsletters:
“Each person you meet influences your mental universe in a way that has the potential to make a substantial impact upon the causality of the intellectual development of an entire species.”
By Abhijit Naskar, Love, God & Neurons: Memoir of a scientist who found himself by getting lost
Index for “AI-powered drug discovery: update part III” 📇
AcuraStem
RNAimmune
AITIA
Empirico
Iambic Therapeutics formerly known as Entos
GT Apeiron Therapeutics
Systems Oncology
Totus Medicines
Valo Health
X-37
Gain Therapeutics
Pharos iBio
🎾 AcuraStem Inc
AcuraStem (2016, US) is a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases—including ALS (Amyotrophic lateral sclerosis) and FTD (Frontotemporal dementia)—by utilizing AI to analyze data from various sources including patient stem cells. iNeuroRx, AcuraStem’s technology, uses living motor neurons (muscle-controlling nerve cells from many amyotrophic lateral sclerosis or ALS patients) and advanced informatics for the identification and development of novel treatments (antisense oligonucleotides).
So far, they have the following programs (with antisense oligonucleotides, ASO) in their pipeline:
PIKFYVE: a novel therapeutic target for ALS and FTD (phase 1/2),
Back in 2018, AcuraStem's co-founders discovered PIKFYVE as a therapeutic target for ALS and FTD in a project led by the co-founder Dr. Justin Ichida and published the results in Nature Medicine. On September 26, 2023, Takeda inked a development and commercialization agreement with AcuraStem to target ALS and other neurodegenerative diseases, of approximately $580M. In return, Takeda gained access to AcuraStem’s PIKFYVE program, which includes AS-202, an investigational antisense oligonucleotide that is an emerging therapeutic target in ALS. Takeda now has exclusive worldwide license to AS-202 as well as to AcuraStem’s other PIKFYVE-directed assets.
Following the $580M deal with Takeda to license the PIKfyve-targeting therapy, on January 30, 2024 AcuraStem announced that it has successfully raised nearly $7M in grant funding for ALS / FTD research from the National Institutes of Health (NIH) and the Department of Defense (DOD), to Accelerate Therapies for ALS and FTD.
UNC13A: a highly genetically validated target for ALS and FTD (preclinical), and
SYF2: a novel target that addresses both gain and loss of function TDP-43 pathology (preclinical).
Around 97% of ALS cases, 45% of FTD cases and 57% of Alzheimer’s cases have the TDP-43 pathology. TDP-43 is a RNA-binding protein normally located in the nucleus, but in patients it is depleted in the nucleus and forms into toxic aggregates in the cytoplasm of neurons.
🏸 RNAimmune Inc
RNAimmune (2020, US, China) is a biopharmaceutical company specializing in discovery and development of messenger mRNA therapeutics and vaccines. They developed a synthetic biology based, AI aided platform that helps them design better mRNAs to invoke the optimal response from our immune system. Using their proprietary Polypeptide Lipid Nanoparticle (PLNP) based carrier, they are able to deliver a wider variety of mRNAs with enhanced intracellular expression.
In particular, RNAimmune holds a global exclusive right to the proprietary PLNP technology for mRNA delivery from Sirnaomics, a leading biopharmaceutical company engaging in discovery and development of advanced RNAi therapeutics. Additionally, RNAimmune possesses various independent proprietary R&D platforms, including the AI and directed neoantigen prediction ALEPVA (Algorithm for Epitope Prediction and Validation) algorithm for nucleic acid sequence design, next-gen lipid nanoparticle (LNP) carrier systems, and circular RNA and self-amplify mRNA platforms. Sirnaomics announced its decision to spin off RNAimmune to develop novel mRNA therapeutics and vaccines back in 2020, with its proprietary know-how large scale cGMP production line and its in-house developed proprietary AI algorithm, ALEPVA, for antigen prediction and vaccine design.
On December 18, 2023, Sirnaomics Ltd (HKG: 2257) announced that its subsidiary RNAimmune has obtained a green light from the FDA to proceed with its Investigational New Drug (IND) application for Phase I clinical trials for RV-1770, an mRNA vaccine targeting the human Respiratory Syncytial Virus (RSV).
RNAimmune has raised a total of $39.4M.
🏏 AITIA BIO
GNS Healthcare, now AITIA BIO (US), uses its Reverse Engineering and Forward Simulation (REFS) or Gemini Digital Twins, namely a causal AI and simulation platform, to analyze RWD and clinical trial data to model patient responses to treatments in silico and has worked with biopharma companies, including Amgen, BMS, Celgene, Johnson & Johnson and Novartis.
Aitia Bio is known for creating Gemini Digital Twins by combining multi-omic and clinical patient data and by using Aitia’s patented causal AI and simulation technology, named REFS. The “Digital Twins” Aitia is creating, can link patients’ characteristics to diverse drug treatments, in order to reveal complex genetic and molecular mechanisms and pathways driving clinical outcomes. For now, Aitia is advancing drug discovery programs in multiple myeloma, prostate cancer, Alzheimer’s Disease, Parkinson’s Disease and Huntington’s Disease.
On March 15, 2023, UCB (EBR: UCB), a global biopharmaceutical company, and Aitia announced an early drug discovery collaboration focused on the discovery and validation of novel drug targets and drug candidates for Huntington’s disease. The collaboration seeks to validate novel drug targets that are causally linked to clinical endpoints in Huntington's disease.
On May 17, 2023, Aitia Entered into Multi-Year AI-Driven Drug Discovery and Drug Simulation Collaboration with Servier for Pancreatic Cancer. The new project with the French pharma group Servier will focus on pancreatic cancer, and builds on a collaboration in the blood cancer multiple myeloma that was started by the two companies last year.
On January 08, 2024, Servier, an independent international pharmaceutical group, and Aitia strengthened their partnership by signing a new contract focused on collaboration in neuroscience. The aim of this cooperation is to leverage Aitia’s Gemini Digital Twins to identify patients most likely to respond positively to Servier’s Leucine-Rich-Repeat-Kinase 2 inhibitor (LRRK2i) in development treatment for Parkinson’s disease.
Aitiabio has 12 investors including Celgene and Cigna Ventures and has raised to date $77.3M.
GNS Healthcare as of January 9, 2023 is Aitia Bio. Aitia—derived from the Greek word for causality—will focus on further leveraging its Gemini Digital Twins to discover the next generation of breakthrough drugs to improve outcomes for patients.
🥍 Empirico Inc
Empirico (2017) is a biotechnology company that combines unmatched expertise in human genetics-driven target discovery with world-class capabilities in siRNA drug discovery, across a broad range of common diseases with unmet medical need. They have built an efficient and integrated drug discovery organization, with fully-internal capabilities spanning computational target discovery, in vitro and in vivo target validation, siRNA chemistry, synthesis and screening, and clinical development. Empirico is headquartered in San Diego, CA with a major second site in Madison, WI, US. Empirico has strategic collaborations with antisense therapeutics firm Ionis Pharmaceuticals and antibody discovery company AbCellera.
The company focuses exclusively on novel, validated targets that they identify as drivers of human disease via in-house human genetics research. Their proprietary platforms utilize leading-edge computational and experimental approaches to both discover novel targets and design therapeutic molecules, and consists of:
Precision Insights Platform™ for the discovery and validation of novel therapeutic targets using human genetics. By combining data from millions of individuals, expertly-curated data models, an ensemble of proprietary statistical, ML and AI algorithms, and a suite of sophisticated applications; the platform enables scientists to systematically generate, interrogate and prioritize high-confidence therapeutic hypotheses. The in silico discovery approaches are combined with proprietary in vitro and in vivo experimental data.
siRCH™ Platform for the design and discovery of clinically-viable siRNA lead candidates. The siRCH process begins with computationally designed and prioritized siRNAs that have favorable properties and are predicted to be active and safe in vivo. Prioritized siRNAs are then synthesized using Empirico’s innovative and proprietary chemistries for hepatic and extrahepatic delivery, and evaluated in their highly-standardized screening workflows. The data generated, as part of the siRNA discovery process, is ingested into the siRCH Platform, enabling continuous learning and improvement of predictions.
In their pipeline their most advanced siRNA program is EMP-012 (IND enabling) for Immunology & Inflammation.
Empirico has raised $91.7M.