In the fast-evolving landscape of biotechnology investments, gene therapies have emerged as the primary drivers, capturing the imagination of investors and industry experts alike. Gene and cell therapies not only hold promise in treating genetic disorders and rare diseases but also constitute the largest portion—one-third—of all investments in the life science field, including biotech, healthtech, novel food, etc. This article explores the current state of gene therapies, their impact on patients, technological obstacles, future perspectives, and the opportunity for small countries to unlock global markets through a regulatory framework for novel drugs. Additionally, it touches on the role of artificial intelligence (AI) in shaping the future of biotechnology.

Gene therapies and patient outcomes

Gene and genetically modified cell therapies are promising drug classes for addressing specific genetic disorders and rare diseases. Some conditions that previously had no available treatments, such as spinal muscular atrophy (SMA, targeted by Zolgensma), or certain inherited blindness (targeted by Luxturna), have witnessed groundbreaking interventions. Similarly, recurring B-cell malignances, treated by various CAR T genetically modified cell therapies, have provided additional years of life for patients who previously had no hope. From the patient’s perspective, these therapies represent a glimmer of hope, offering the potential for transformative treatments that were once considered beyond the realm of possibility.

While gene therapies hold great promise, they are still in the early stages of development and face challenges related to safety, accessibility, and regulatory approval. Therefore, all currently available gene- and genetically engineered cell therapies, and most of those nearing market readiness, center on Orphan Drug Designation Application. These therapies mostly address hopeless and life-threatening conditions, in which the potential benefit for the patient clearly outweighs the cost and potential risks posed by the novel, almost experimental therapy. This approach focuses on being the last line of treatment with no other available options for improvement or halting the progression of the patient’s condition. Consequently, and due to the cost, the current drugs on the market affect a relatively small number of individuals.

Ongoing clinical trials: Expanding the horizon

The dynamic interplay between gene therapies and orphan drugs exemplifies the evolving landscape of medical genetics research and treatment modalities. Both avenues contribute to the pursuit of innovative solutions for challenging medical conditions. The designation of a drug as an orphan drug can provide various incentives for pharmaceutical companies to invest in high-risk research and development for these conditions. These incentives may include market exclusivity, tax credits, and research grants.

There are over 2000 therapies in various stages of preclinical research or clinical trials. Beyond rare diseases, these trials seek to broaden the application of gene therapies to more prevalent conditions like certain cancers, neurodegenerative disorders, and cardiovascular diseases. However, current knowledge about the risks, coupled with our incomplete understanding of the long-term effects of engineered genes and mechanisms of action of many diseases, often trims the list once clinical trials commence.

During the clinical stage, the risk-benefit profile in real human conditions is critically assessed, allowing only those treatments to progress that demonstrate viability through significant improvement with no imminent serious side effects. Additionally, the high cost of the novel therapies set even elevated demands for the observed clinical benefits to be justified. Therefore, clinical results and economic viability criteria are unforgiving, significantly reducing the current list of drugs progressing through the development process as they approach the actual market.

Nevertheless, as the trials progress, the amassed wealth of knowledge derived from these trials, whether failed or successful, along with a growing scientific interest in the field, significantly enhances the likelihood that gene therapies may evolve into mainstream treatments for a broader spectrum of diseases.

Technological obstacles: navigating challenges

Currently, the success of gene and genetically engineered cell therapies in development is haunted by three main challenges:

  • Challenge 1: Mechanisms not clear. The insufficiency of knowledge about the genetic mechanisms of complex diseases and the effects arising from genetic modifications through gene therapies.
  • Challenge 2: It is expensive. The absence of cost-effective and scalable production and delivery methods.
  • Challenge 3: Addressing safety is uncoherent. The lack of long-term safety and efficacy profiles, coupled with the absence of a coherent methodical framework to preemptively assess these questions before delivering therapies into humans.

The growth of the gene therapies market remains limited when even a single challenge is inadequately addressed.

Solving the Challenge 1 and Challenge 2 holds much promise through fundamental scientific research that broadens our understanding of the mechanisms of life. Especially with the Challenge 1, the downside is that quite often, planning the outcome of such research is impossible, even when seemingly “right” solutions or “effective” strategies are hypothesized in advance.

In this sense, any laboratory, research institution, or even startup company with well-conducted experiments and a bit of luck could contribute valuable solutions. It is crucial to emphasize that addressing the inherent risk in Challenge 1 necessitates a sufficiently wide array of research, sometimes even overlapping, conducted in the field. Today, major attention from the corporal and private investments sector is currently focused on solving the Challenge 2. Therefore, governmental push is mostly expected for the Challenge 1 and Challenge 2.

Despite the Challenge 1 being addressed through major research programs by many governments or corporations, the Challenge 3 is somewhat underinvested, receiving limited attention from both sides. That means the current portfolio of research and development methods available, the experimental practices expected by regulatory authorities, and conventional expectations for drug safety profiles do not align as seamlessly as they do for low molecular metabolized drugs. This misalignment poses significant risks, leading to delays in launching novel gene therapy products today and casting uncertainty over their long-term safety in the future. It underscores the timely and safe introduction of innovative gene therapies.

Future perspectives: Unlocking the market potential, ethical & social considerations

Nevertheless, for even relatively small countries, such as Singapore or Estonia, addressing the Challenge 3 can become a strategic enabler for the gene therapies market and provide a tremendous acceleration of the sector. The first-mover advantage in proposing clear framework in this area will result in many benefits for the country’s healthcare economy:

  1. It will facilitate rapid access to novel drugs for local patients at a much lower cost by granting them access to clinical or long-term studies, or by opening a unique position for price ceiling negotiations between the government and corporations.
  2. Additionally, it will attract novel drug development programs from all over the globe to the country, coupled with a rapid influx of investor capital to support the development of emerging pipelines.
  3. Lastly, it will contribute to the rapid growth of the private healthcare market for foreign patients seeking help from the first enabling countries.

Therefore, addressing Challenge 3 can become a true enabler for the rapid development of the sector in the country.

To effectively address Challenge 3, the lead role should be taken by the government, targeting research at the interface of fundamental research, clinical practice, and corporate drug development programs. Expecting rapid solutions by placing the responsibility solely on one counterpart is futile. The ultimate target is to find a balance between the interests of society and individuals, public healthcare and the profitable private sector, technological and ethical considerations.

Researchers are already exploring genetic interventions for anti-aging purposes, primarily at the level of public fundamental or early preclinical corporal research. If such potential could be realized in therapies, critical issues of access and affordability would emerge and must be addressed to prevent healthcare disparities and economic burdens. Currently, even most orphan drug designation gene therapies on the market cost more than $1.5 million per patient.

Furthermore, discounting all patent-related or R&D cost-related components, there are currently no viable technologies to enable any gene therapy below $50,000 per single dose per patient, posing a significant challenge for the future development of the generics market for these therapies. Another important issue, germline editing, with its potential to shape future generations, demands careful ethical and methodical scrutiny to avoid unintended consequences or regulatory deadlock later.

Therefore, in addition to addressing methodical or technological challenges, the regulatory framework should encompass the ethical and potential societal impact of novel drugs. It should also facilitate early communication to generate widespread understanding in society about the reality of the future ahead. Be the coherent action taken at the Challenge 3, the market readiness and uninterrupted growth of the sector will be achieved.

In summary

The future of biotech investments hinges on the continued evolution of gene therapies and the integration of novel technologies into biological research standards. While gene therapies hold immense promise, stakeholders must navigate regulatory, ethical, technical, and societal considerations. As the field advances, novel methodical interfaces, such as gene editing for longevity and AI as a tool for biologics, pose challenges for the regulatory framework. Such frameworks are central to unlocking new frontiers and market potential. Careful attention to experimental validation and data quality, as well as clear communication, will be paramount.

In this dynamic landscape, small countries such as Singapore and Estonia might achieve catalyzing potential by providing relevant, consistent, and innovation-driven regulatory frameworks to unlock the full market potential of biotechnological advancements.

Dr. Rainis Venta is a biomedical engineer by training and was recently apponted as the (Deep) Technology Attaché of Estonia in Singapore. The appointment is timely with Singapore’s own focus on deep technology and is in line with The Embassy of Estonia in Singapore’s and The Estonian Business Hub’s ongoing commitment to strengthening Estonia-Singapore ties.

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