28-29 - MEDICINE: Taking flight

“If we worked on the assumption that what is accepted as true really is true, then there would be little hope for advance.” Orville Wright

The COVID-19 pandemic ushered in an unprecedented level of collaboration among key stakeholders in the pharmaceutical industry, propelling the rapid development and production of three effective vaccines for the US market. Data transparency and sharing played a major role in that success.

The same collaborative approach – driven by data sharing – should now be carried forward to create the next generation of genomic medicines. These are treatments that precisely modify DNA, RNA or their cellular machinery to treat or prevent disease.

This emerging class of treatments includes the two approved mRNA vaccines against COVID-19, Moderna’s Spikevax and Pfizer/BioNTech’s Comirnaty. It also encompasses gene therapies and genetically modified cell therapies, such as CAR-T treatments for cancer.

Genomic medicine has reached what we like to call its ‘Kitty Hawk’ moment. What does that mean? The Wright Brothers were committed to their vision of air travel and that motivated them to continue building their pioneering Kitty Hawk Flyer, while larger organisations such as government agencies pursued human flight without much-needed investments, thus making little progress.

Similarly, the sequencing of the human genome generated excitement in 2000, but the only scientists at that time who were committed to the vision of genomic medicine were hampered by limited resources and interest. And like the Wright brothers – who had to build everything from scratch – few tools and infrastructure existed then to support the development of gene-based therapies.

Now there is a wealth of tools to support genomic medicine manufacturers, perhaps even enough to move the field beyond its Kitty Hawk moment. We are perfecting drug-delivery vehicles like lipid nanoparticles, for example. And we’re streamlining production methods to cut costs. But the importance of open data in moving the industry beyond this moment cannot be overstated.

With COVID, the biopharmaceutical industry fostered collaboration among multiple stakeholders. We saw companies that are direct competitors producing each other’s vaccines. We saw government regulators stepping up and collaborating closely with drug developers to compress development and manufacturing timelines. Data sharing was key to those collaborations.

Why is data transparency and sharing so critical to advancing genomic medicine? Because we are in the age of programmable, digital biology, and the more data scientists can share about what they’re learning, the faster we can develop treatments to address the greatest unmet needs in medicine.

When it comes to creating a more open approach to data sharing, pharmaceutical developers should look to the software industry for inspiration. It has an effective open-source system that fosters innovation. Not everything is open source, to be sure, but there is a set of robust software tools that everybody in the software industry can use.

One major asset genomic medicine has that the Wright brothers did not is the internet. While every industry benefits from the unprecedented access to information and opportunities for collaboration the online world offers, the most complex endeavours reap the most benefits.

Biopharmaceutical researchers across the globe in academia, industry and government are working together virtually, with many companies identifying ‘pre-competitive’ scientific areas where they can collaborate with government and academic labs to avoid reinventing the wheel.

This is a good start, but we need to do better. We need to create a ‘Goldilocks zone’ of data sharing – information that biopharma developers can share in a way that lowers barriers to innovation, while at the same time allowing them to retain their key competitive advantages.

For example, researchers could share biodistribution statistics associated with a drug payload that’s delivered by a particular viral vector or polymer without revealing the cargo’s genetic sequence. Companies could share advances in analytical techniques that are specific to therapeutic classes but not to any individual product.

They could also provide access to the data from the many approaches they tried that didn’t work. This data related to research failures would inform future work in the same therapeutic area, as well as approaches other scientists might be considering for treating emerging diseases.

To widen this Goldilocks zone, government institutions and regulators should provide more incentives for contributing, aggregating and analysing data across the industry. The end result will be significant reductions in development timelines, production costs and the duration and costs of clinical trials.

Now that the genomic medicine field is edging beyond its Kitty Hawk moment, many companies are being formed to address the most significant challenges the industry is facing.

Some are developing more efficient and safer viral vectors, while others are working on improving gene-editing technologies. Existing companies are expanding capacity, new companies are forming that are more vertically integrated, and these new entities are investing in better processes and methods.

Once the Wright brothers showed the potential of flying machines, others joined, expanding innovation and creating a market for the tools and services associated with flying machines. Wilbur Wright noted the scaling effect of demonstrating that the impossible is indeed possible when he said, “What one man can do himself directly is but little. If he can stir up ten others to take up the task he has accomplished much.” Similar momentum is being generated in genomic medicine, driving down the costs of research and production.

Now we must embrace data transparency to build on this strong foundation, so we can address a broader range of indications and bring cutting-edge therapies to many more patients.

Taking flight

Perceived wisdom

Software industry

Goldilocks zone

Taking off