The biotech and pharmaceutical industries are entering 2024 amid a wave of innovation and technological advancement.
The biopharma field is increasingly influenced by the advent of cutting-edge technologies such as artificial intelligence (AI), organ-on-a-chip systems, and gene editing therapies promising more personalized and precise treatments for a variety of conditions.
For instance, AI’s role in analyzing complex biological data is streamlining the identification of therapeutic targets and optimizing drug design.
Organ-on-a-chip technology offers a more efficient and ethical alternative to traditional drug testing methods, potentially reducing development times and costs.
Meanwhile, gene editing therapies, e.g. for managing high cholesterol, represent a significant leap towards treating diseases at their genetic roots, offering new hope for previously intractable or poorly controlled conditions.
In this article we highlighted a number of companies representing a wide range of technologies, from the first AI-generated drug entering stage II clinical trials to protein design in silico, offering insights into their novel approaches and the potential impact of their work on the future of healthcare.
Generate Biomedicine is a biotechnology company leveraging the power of computational methods to design proteins possessing a desired function that have never existed in nature.
Company’s technology relies on two main elements. The first involves generating new proteins de novo, where a computer designs sequences that can bind to specific targets without prior knowledge of the binding process, surpassing the limitations of the immune system’s natural output. The second focuses on optimization, transforming the concept of a computationally designed protein into a practical therapeutic. To achieve this, Generate:Biomedicines employs an optimization suite that refines native proteins into effective therapeutics for specific targets.
Since the company’s launch in 2018 they managed to secure substantial funding for achieving their goals. In their latest Series C funding round in September 2023, Generate:Biomedicines secured $273 million in Series C funding, one of the largest for a biotech firm last year, elevating their total equity financing to nearly $700 million since 2020.
While the cost of clinical trials in the world is plummeting following Eroom’s law, Vial, a San Francisco-based contract research organization (CRO), made its goal to reduce the cost of clinical trials by an order of magnitude.
Vial aims to streamline the clinical trial process through integration of eSource, Electronic Data Capture, and electronic Patient Reported Outcomes into a single system. This platform is designed to be modern, intuitive, and responsive, enhancing the efficiency of trials while ensuring they remain cost-effective for biotech sponsors. The company operates across various therapeutic areas, including dermatology, ophthalmology, oncology, gastroenterology, neurology, cardiology, medical devices, rare diseases, and digital therapeutics.
Currently, Vial is running 12 clinical trials, two of which are in Phase III. Trials the company is involved in range from real-world efficacy study of an eye disease to trials for tropical diseases in partnership with the nonprofit Medicines Development for Global Health. You can learn more about how Vial is operating in this Century of biotech blog post.
Founded in 2018 in Boston, Verve Therapeutics aims to address the ongoing challenge of cardiovascular disease, which, despite advancements in treatment and care, continues to be the primary cause of death globally.
In the summer 2023 the company presented a novel technique to help prevent heart disease that has been highlighted in Science. This technique involves editing the genetic code within the body to lower levels of “bad” cholesterol, potentially offering a long-term solution for individuals with high cholesterol due to genetic predispositions.
This method involves an infusion of a base editor designed to inactivate a liver protein known as PCSK9 that plays a key role in cholesterol regulation. The clinical trial demonstrated that this approach could efficiently target and edit genes within the liver, marking a shift towards using gene editing technologies for common health issues like high cholesterol. This was the first instance of using a CRISPR-derived base editing tool in humans to treat a disease, suggesting a potential for broader applications beyond rare genetic conditions.
However, the technology’s safety and cost implications remain areas of concern. The trial revealed that while the base editor successfully reduced PCSK9 and LDL cholesterol levels in patients, there were instances of serious cardiac events, raising questions about the treatment’s safety profile. Additionally, the high cost of gene therapies could limit access to this treatment. Despite the challenges, Verve Therapeutics plans to expand its trials and aims to make the treatment more accessible than existing gene therapies.
Concerto Biosciences is pioneering the field of microbial community restoration. Founded in 2020 by a team of innovators from MIT and the Broad Institute, Concerto is developing kChip technology to measure and understand millions of microbial interactions.
The company aims to redefine humanity’s relationship with microbes by moving away from traditionally employed microbial eradication towards a model that seeks to understand, preserve, and enhance beneficial microbial ecosystems.
Concerto is collecting data on the complex networks of microbial life to develop “ensembles” – synergistic combinations of microbes aimed at healing damaged microbial communities. With over 6 million microbial interactions measured, the company has established a significant foundation for its research, including a comprehensive human skin microbe biobank.
This technology has already facilitated the creation of their first product, Ensemble No.2 (ENS-002), targeting the skin microbiome to alleviate the symptoms of eczema.
Insilico Medicine is a biotechnology company pioneering the use of generative artificial intelligence (AI) and deep learning for drug discovery and development.
Founded in 2014 by Alex Zhavoronkov, the company aims to leverage AI to accelerate the process of identifying promising drug candidates, predicting their molecular properties, and designing novel molecules for a wide range of diseases.
Insilico has developed several AI-driven platforms, among which are PandaOmics for target identification and Chemistry42 for designing novel drug compounds, and inClinico for modeling clinical trials. The platforms are combined in an end-to-end Pharma.AI engine.
In early 2023, INS018_055, a novel antifibrotic small molecule inhibitor by Insilico, received positive topline data in Phase I yielding consistent results, demonstrating favorable safety, tolerability, and pharmacokinetics (PK) profiles, supporting the initiation of the Phase II study.
The trials aim to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of INS018_055 in patients with Idiopathic Pulmonary Fibrosis (IPF) over a 12-week oral dosage regimen. The case of Insilco highlights the potential of generative AI in drug development for the unmet medical needs.
92% of all clinical trials are poised to fail.
To address this challenge, Quris AI combined the power of artificial intelligence and organ-on-a-chip systems into a unique Bio-AI Clinical Prediction Platform.
This platform integrates high-throughput 3D and multi-organ technologies, real-time sensing, and stem-cell genomic diversity with advanced machine learning to improve the prediction of clinical safety for drug candidates.
Quris AI’s technology uses interconnected miniaturized human organs (for example, the liver and brain), to simulate human metabolic responses more accurately than traditional organ-on-a-chip models. This system is capable of generating extensive data on drug metabolites in time-dependent manner through integrated sensors, aiming to enhance the accuracy of safety predictions.
By applying AI to analyze the large datasets generated by its platform, Quris takes a step beyond the capabilities of other organ-on-a-chip technologies allowing for the automated testing of drugs. The platform has demonstrated high sensitivity and specificity in detecting liver toxicity and offers scalability and cost-effectiveness advantages. With 29 granted and pending patents, Quris is positioned to contribute to the shift towards non-animal testing methods in drug development, supported by recent legislative changes like the US FDA’s Modernization Act 2.0.
Dallas-based Lantern Pharma Inc. integrates artificial intelligence (AI) with oncology to enhance the efficiency of clinical trials via biomarker-led clinical trial design.
Since its creation in 2014, the company aims to improve standards of care for cancer patients by expanding therapeutic indications space, identifying combination therapy opportunities, and designing better clinical trials with precision-based patient selection strategies.
To achieve these goals, Lantern developed a ground-breaking tool RADR®, an AI-driven platform that exploits machine learning for identifying mechanisms of action (MoA), driving preclinical development of oncology assets, identifying novel therapeutic indications, and discerning and predicting patient responses to specific drugs. RADR® analyzes over 25 billion clinical data points, more than 154 drug-tumor interactions, and 130,000+ patient records across 17 databases. This end-to-end process transforms multi-omics data into an optimal model that predicts drug responses, generating candidate biomarkers and aiding in patient stratification for clinical trial design.
Lantern’s portfolio now includes four drug candidates across two Phase 2 programs, an antibody-drug conjugate program across 12 cancer indications, and several partner programs, illustrating the platform’s potential in enhancing clinical therapeutic pipelines in the industry. Through this data-driven method, Lantern has also streamlined their preclinical drug development process, managing to reduce the average time from AI-derived insights to first-in-human clinical trials to 2-3 years at a budget of $1.0-2.0 million per program.
Read also: How AI Empowers Precision Oncology
Founded in May 2021, San Francisco-based Atomic AI is combining the power of machine learning with structural biology to discover novel RNA therapies. Atomic AI’s proprietary AI-driven 3D RNA structure engine, known as PARSE, generates RNA structural datasets, integrating machine learning foundation models with large-scale, in-house experimental wet-lab biology to unveil functional binders to RNA targets. The company’s technology has the ability to predict structured, ligandable RNA motifs at unprecedented speed and accuracy, a key barrier to current approaches to RNA drug discovery.
In December 2023, the company unveiled a novel platform ATOM-1 representing a large language model (LLM) that leverages chemical mapping data to predict the structure and function of RNA. ATOM-1 aims to address challenges associated with designing RNA therapeutics by optimizing key characteristics of RNA modalities (stability, toxicity, and translational efficiency) with a minimal amount of initial data points.
Atomic AI has successfully secured $42 million in funding across two rounds of investment, with the most recent capital infusion coming from a Series A round in January 2023. The company boasts support from 14 investors, including notable recent contributors Nat Friedman and Neal Khosla.
Kymera Therapeutics, Inc. was one of the first companies that saw a therapeutic potential in a naturally occurring phenomenon of protein degradation. Today, Kymera is a clinical-stage biopharmaceutical company based in Massachusetts advancing a new class of small molecule medicines using targeted protein degradation (TPD).
This technology addresses the root cause of the disease by selectively eliminating disease-causing proteins via the body’s natural cellular recycling system.
Kymera creates heterobifunctional small molecules that redirect natural protein degradation by engaging enzymes called E3 ubiquitin ligases to recruit disease-causing proteins and place a chain of ubiquitin molecules on the recruited protein.
The ubiquitin tag is recognized by the cell’s recycling systems—the proteasome—to selectively break down the protein. These molecules are catalytic, performing this tagging action multiple times within the cell. For hit identification, the company uses such techniques as DNA encoded libraries, fragment and X-ray based screenings, machine learning and artificial intelligence hit-validation, structure and pharmacokinetic/pharmacodynamic-centric enabled lead optimization. Kymera focuses on therapeutic areas that cannot be addressed by traditional approaches, targeting undrugged proteins and broad pathways with strong genetic and clinical validation in a disease-agnostic way.
Currently the company has 5 drug candidates in the fields of oncology and immunology, 3 of which are in phase I of clinical trials, and 2 in phase II. The most recent achievement of Kymera was FDA fast track designation for KT-333 for treating R/R CTCL and PTCL. KT-333, a STAT3 degrader, targets inflammatory, autoimmune diseases, and various cancers. It also has orphan drug designation for CTCL and PTCL. A Phase I trial is assessing KT-333’s safety and efficacy in adults with R/R leukemias, solid tumors, and lymphomas.