Drug discovery in space is emerging as one of the most exciting frontiers in pharmaceutical innovation, offering new opportunities to identify, develop and manufacture improved medicines.
Pharmaceutical companies, biotechnology start-ups and academic institutions invest enormous resources in identifying new drug treatments. Whilst considerable attention is often directed towards the discovery of new active ingredients, the manner in which those ingredients are formulated and delivered to patients can be equally important.
A significant number of drug candidates fail commercially, or perform below their full potential, not because the active molecule lacks efficacy, but because their solid-state form is unsuitable as a pharmaceutical. A single compound can exist in multiple crystalline forms, known as polymorphs, each with different physical and pharmacokinetic properties, which can have a profound impact on clinical performance and commercial success. As a result, life science companies are continually seeking new crystal forms of active ingredients that offer enhanced pharmaceutical performance, stability and ease of manufacture.
At the same time, there is a growing demand for effective formulations that are easy to store, transport and administer. This reflects a clear trend towards treatments that can be used by patients at home rather than in hospitals or specialist clinics.
This is particularly relevant in the field of biologics. Many modern cancer therapies, including monoclonal antibody treatments, are administered by intravenous (IV) infusion under medical supervision, often requiring patients to spend several hours in hospital. These repeated visits place a considerable burden on both patients and healthcare providers. One of the major goals for healthcare providers is therefore to transition to treatments based on self-administered formulations, whether as oral tablets and pills, subcutaneous injections or long-acting therapies.
With this in mind, researchers are turning to an unlikely environment in the search for improved medicines: space.
Why drug discovery in space matters for modern drug formulation
Monoclonal antibodies have transformed cancer immunotherapy, as well as the treatment of autoimmune disorders and a range of other serious diseases. Their success, however, comes with significant formulation challenges.
Monoclonal antibodies are very large molecules, much larger than conventional small-molecule drugs. At high concentrations, they tend to interact with one another, which can increase viscosity, promote aggregation, and complicate formulation. As a result, these drugs are often supplied as liquid formulations that require administration by hours-long IV infusion in a hospital.
Developing concentrated, stable antibody formulations suitable for quick self-injection could therefore represent a major advance in pharmaceutical science. Such formulations would not only improve patient convenience but could also reduce healthcare costs and increase access to treatment.
Drug discovery in space through microgravity manufacturing
One exciting approach currently being explored is the use of microgravity to develop improved pharmaceutical formulations.
On Earth, gravity affects the way crystals form and grow. Convection currents and sedimentation can influence crystal structure and introduce defects that limit the quality and uniformity of the final product. In microgravity, these effects are greatly reduced, allowing molecules to assemble under different conditions.
Researchers have long recognised that crystals grown in space under microgravity conditions can be larger, more uniform and more ordered than those produced on Earth. These characteristics may prove particularly valuable in the manufacture of complex biological medicines.
Merck's work on its blockbuster cancer medicine Keytruda® demonstrated the potential value of microgravity-based crystallisation. Experiments conducted aboard the International Space Station (ISS) helped researchers investigate methods for producing more uniform protein crystals, providing insights that were subsequently applied to formulation development on Earth. Building on this work, Merck managed to secure FDA approval for a quick injectable version of Keytruda® that can be administered in about one minute rather than a 30-minute IV infusion.
Among the companies seeking to commercialise this approach is the UK start-up BioOrbit. The company is developing a microgravity-enabled protein crystallisation platform designed to produce ultra-pure protein crystals that could form the basis of next-generation biologic medicines.
Central to this effort is Box-E, an autonomous manufacturing reactor capable of operating in orbit without crew intervention. The modular design is intended to support scalable production and could provide a route towards routine pharmaceutical manufacturing in space.
Protein crystallisation can be notoriously difficult. Unlike small molecules, proteins have complex three-dimensional surfaces and are typically sensitive to pH, salts, temperature, and impurities. Obtaining uniform, stable crystals suitable for manufacturing can therefore be challenging. This is why protein crystallisation has historically been used more as an analytical or structural biology tool rather than as a mainstream pharmaceutical manufacturing process.
Microgravity may help overcome some of these limitations. By enabling the formation of highly ordered crystals, it may be possible to create formulations with improved stability, lower viscosity and higher concentrations while still maintaining injectability.
Although manufacturing in orbit remains expensive, reusable rockets have lowered the cost of access to space and opened up a range of in-space activities. If space-based manufacturing can facilitate the development of treatments that reduce hospital visits and simplify administration, the economic case may become increasingly attractive.
BioOrbit recently completed a record-setting International Space Station mission, producing protein crystals at a scale reportedly one to two orders of magnitude greater than previous comparable experiments. Clinical applications remain several years away, but the company believes commercial products could emerge within the next decade.
The search for new crystal forms
The opportunities extend beyond biologics.
Another company, SpaceMD, is exploring how microgravity can be used to discover novel crystal forms of pharmaceutical compounds. SpaceMD has developed a compact automated crystallisation platform known as PIL-BOX, which functions as a miniature laboratory capable of conducting crystallisation experiments while in orbit.
Many drugs can crystallise as different polymorphs with different crystal structures. A drug’s crystal structure impacts its properties, such as dissolution rate, solubility, bioavailability and stability. Identifying an improved polymorph can therefore significantly enhance the performance of an existing active ingredient.
Microgravity can alter how molecules assemble into crystals, which can lead to different crystal polymorphs forming in space compared to on Earth.
Because molecules can assemble differently in microgravity, entirely new polymorphs may be obtained that are difficult, or in some cases impossible, to produce on Earth. Companies such as SpaceMD hope to exploit this phenomenon to discover new pharmaceutical forms with superior properties and improved manufacturability by growing crystals in space.
The future of drug discovery in space: Building an orbital pharmaceutical industry
For pharmaceutical manufacturing in space to move beyond experimental programmes, a supporting industrial infrastructure will be required.
Varda Space Industries is focused on the development of commercial space infrastructure by building a system that allows for pharmaceuticals to be manufactured in space. The company describes its spacecraft as autonomous orbital factories capable of manufacturing compounds in microgravity before returning them to Earth in dedicated re-entry capsules.
Whilst many products are too bulky or low-value to justify manufacturing in orbit, pharmaceuticals are different. Drug substances often have extremely high economic value relative to their size and weight. The quantity of crystalline active pharmaceutical ingredient needed to treat hundreds of millions of patients can fit into a very small volume, making launch and recovery costs potentially economically viable.
Varda believes this economic reality could allow pharmaceuticals to become one of the first commercially viable large-scale manufacturing sectors in space. If so, the industry may provide an important foundation for broader industrial activity in low Earth orbit.
Intellectual property considerations for drug discovery in space
The prospect of manufacturing medicines in space inevitably raises interesting questions about intellectual property.
On Earth, patents are territorial rights, meaning that they are generally enforceable only within the country or region in which they are granted. One of the main challenges in enforcing patents in space is the fact that outer space is not subject to the jurisdiction of any one country. This raises important questions when patented technology is used beyond national borders, including in outer space.
Under the Outer Space Treaty (OST) of 1967, outer space and celestial bodies are not subject to national appropriation or claims of sovereignty. However, space law provides that a spacecraft generally remains under the jurisdiction and control of its state of registry, rather than the physical location of the activity. As a result, the laws of that state, including its patent laws, may apply to activities carried out on board the spacecraft. For instance, the use of a patented invention aboard a US-registered spacecraft could potentially infringe a US patent, whereas the same activity carried out aboard a spacecraft registered elsewhere may fall outside the scope of those rights.
In the case of the ISS, which consists of modules, each module is registered to different partner states. Each module is effectively treated as an extension of the territory of that state, meaning that the national laws of that state, including its patent laws, generally apply within that module.
Nevertheless, as commercial activities in space continue to expand, significant legal and practical questions remain regarding the scope and enforcement of patent rights in orbit and beyond.
Despite these challenges, innovation in this sector remains highly patentable. Protection may be available for new formulations, crystallisation processes, manufacturing systems, spacecraft-based production equipment and methods for producing improved pharmaceutical products.
From a strategic perspective, applicants should carefully consider the nature of their inventions. Apparatus claims directed to manufacturing equipment may be easier to enforce through activities taking place on Earth, including the manufacture, launch and operation of the relevant hardware. Process claims may present more complex enforcement issues if the critical steps occur in orbit.
In some cases, however, protection directed to the resulting pharmaceutical product may offer a more practical route to enforcement once the product returns to Earth and enters commercial supply chains. Patent protection will need to account for not only where medicines are ultimately sold and used, but also for where manufacturing, launch and recovery activities occur, as well as the jurisdiction in which the relevant spacecraft are registered.
The commercialisation of space-based pharmaceuticals may still be in its early stages, but the convergence of biotechnology, advanced manufacturing and the space sector is creating fascinating opportunities. For innovators operating at this frontier, a well-considered IP strategy could prove just as important as the science itself.
If you are exploring novel drug formulations, manufacturing processes or drug delivery technologies and would like to discuss patent protection strategies, please contact Fabian Carson in our life sciences team.
