Rise of the Biotech Platforms

In the ever-evolving landscape of biotechnology, the paradigm of investment and innovation is undergoing a significant transformation. Traditionally, biotech investments gravitated towards university laboratories where individual scientists pursued unique and often groundbreaking research. However, a notable shift is underway: the focus is increasingly on platform technologies. From 2019 to 2021, VC companies invested more than $52 billion in therapeutic-based biotech companies globally and two-thirds of that went to start-ups with platform technologies.

These platforms, offering a systematic approach to drug development, promise a revolutionary way forward. From an investor perspective, the platform model is far more attractive, offering patentable technology which has the potential to produce ongoing innovation. This article explores the precise nature of these technologies, and the investment outlook for both platforms and traditional drug development.

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Explaining Platform Technologies:

Platform technologies in biotechnology represent a framework or system that can be repeatedly used to develop new products or processes. These platforms range from scalable systems for testing samples in live cells to advanced AI algorithms capable of modeling target compounds to identify potential uses. Their appeal lies in their patentability and scalability. Unlike traditional one-drug-at-a-time approaches, these platforms can potentially churn out multiple new molecular entities, offering a continual pipeline of innovation.

Specific types of platforms include the following:

Advanced Cell Therapies:

The evolution of cell therapy, often termed "Cell Therapy 2.0," has made significant strides since 2017. Initially centered around CAR T cell therapies for hematologic malignancies, this field is now expanding to address a broader range of diseases, including solid tumors. Despite remarkable successes, challenges like safety concerns and the complexity of treatment processes persist. Innovations in this space include focusing on innate immune cells like NK cells for better tumor penetration, employing synthetic biology for more precise control of cell therapies, and developing in vivo cell therapies that reduce logistical and manufacturing complexities. These advancements, backed by increasing venture capital investments, are pushing the boundaries of cell therapy, aiming to make it more effective and widely applicable.

Fate Therapeutics: Pioneering the development of programmed cellular immunotherapies for cancer and immune disorders. They use engineered immune cells, tailoring treatments to individual patients, a cutting-edge approach in advanced cell therapies.

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Next-Generation Gene Therapies:

Gene therapies are rapidly transforming treatments for genetic disorders, with around 400 therapies in development as of 2021, potentially making up 20% of new product launches by 2025. Despite advances, limitations in gene editing like CRISPR-Cas9's range and safety concerns remain. To address these, biotech companies are exploring RNA-based editing, novel nucleases with improved efficiency, and non-nuclease editing technologies for more precise and diverse genetic modifications. These next-generation strategies, gaining significant venture capital, aim to expand the potential and safety of gene therapies.

Editas Medicine: As a leader in next-generation gene therapies, Editas Medicine leverages CRISPR gene editing technology. They focus on developing treatments that correct genetic defects at their source, targeting a range of genetic disorders.

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Precision Medicine:

Precision medicine uses diagnostics and analytics, including AI, to tailor treatments based on individual genetic, environmental, and lifestyle factors. While it has led to specific treatments for certain gene mutations, challenges remain. These include the limitation of first-generation diagnostics to only known biomarkers, the need for identifying unique disease subpopulations, and the difficulty of translating genetic data into clinical decisions. Emerging biotech companies are focusing on early disease detection using advanced tools, novel biomarker discovery, and precision population health to address these challenges and advance the field.

Foundation Medicine: Specializing in precision medicine, Foundation Medicine offers comprehensive genomic profiling. This approach helps identify targeted therapies and immunotherapies for cancer patients, tailoring treatments based on individual genetic profiles.

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Machine Learning-Enabled Drug Discovery:

Machine learning (ML) in drug discovery is advancing rapidly, offering the potential to accelerate the development of new drugs through simulations and protein structure predictions like AlphaFold. However, challenges such as limited data quality, non-generalizable models, and complex algorithms still exist. To overcome these, startups are focusing on areas like target identification, rational drug design, and lead validation using sophisticated ML techniques. These efforts aim to enhance the accuracy and efficiency of drug discovery, although they still face developmental and financial challenges.

Exscientia: Exscientia stands at the forefront of AI-enabled drug discovery. They use machine learning to analyze vast data sets, aiming to speed up the discovery and development of new drugs more efficiently.

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Strategies for 'Undruggable' Targets:

Conventional drugs often target proteins, but many disease-related proteins are considered 'undruggable'. Recent biopharma advances focus on overcoming this, attracting renewed investor interest. Challenges include drug resistance and limited knowledge of disease biology. Biotech startups are developing new approaches to target these proteins, including finding new small-molecule binding sites, exploring protein degradation techniques like PROTAC, and identifying novel disease targets by studying disease-resistant populations. These innovations aim to expand treatment options for hard-to-treat diseases.

Relay Therapeutics: Focuses on targeting 'undruggable' proteins. They employ a unique approach that involves understanding protein motion to discover new therapies for challenging diseases.

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Novel Drug Delivery Methods:

Drug delivery technology is evolving, with lipid nanoparticles like those in COVID-19 vaccines being key. Challenges include delivering large molecules and limited tissue targeting. Biotech startups are focusing on improved capsids using machine learning, biological vehicles like exosomes for safety, and enhanced nanoparticles for wider tissue reach. This progress is crucial for the efficacy of over 400 RNA-based therapies in development.

Capsida Biotherapeutics: Innovating in the field of drug delivery, Capsida Biotherapeutics uses a capsid engineering platform to develop targeted gene therapies, enhancing the precision and safety of drug delivery.

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Investor Outlook:

The allure of these technologies to investors is clear. The traditional model, dependent on individual scientific pursuits in university settings, is often seen as less predictable and slower to yield tangible products. In contrast, platform technologies promise a more streamlined and economically attractive pathway. They are not just about producing single drugs; they are about creating the means to produce many drugs, making them a more lucrative prospect for investors seeking substantial returns.

However, this shift raises significant concerns. The primary downside is the potential for overlooking beneficial treatments that do not fit into these systematic, platform-based approaches. Platform technologies, by their very nature, tend to focus on areas where high throughput and scalability are possible. This focus can inadvertently marginalize research areas that are less conducive to such approaches but are nonetheless vital. For example, rare diseases or complex conditions that require more nuanced, individualized research might fall by the wayside.

The solution doesn't lie in discarding the new in favor of the old or vice versa. Instead, a balanced approach is crucial. Investors and policymakers must recognize the value of both methodologies. Strategies to ensure a diversified investment landscape could include incentivizing investments in traditional research, especially in areas where platform technologies are less applicable. Collaborative models where platform technologies aid traditional research efforts could also be explored, leveraging the strengths of both approaches.

Platform technologies undeniably represent a leap forward in drug development, offering efficiency and a continuous pipeline of potential new therapies. However, the biotech sector must tread carefully, ensuring that this new model of innovation and investment does not eclipse the invaluable contributions of traditional scientific research. A symbiotic relationship between the old and the new, where each complements the other, could be the key to a future where no potential treatment, no matter how unconventional or challenging, is overlooked.

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Yasir Hassan

The Biotech Investor