Since the 1990s, we’ve seen an expansion in the gene therapy landscape, which is expected to continue growing in the coming years. (Cell Stem Cell) The global gene therapy market was valued at $6.0 billion in 2020 and is projected to reach $46.5 billion by 2030, growing at a CAGR of 22.8% from 2021 to 2030. (AMR)

Gene therapies can potentially target a wide range of diseases, including cancer, infectious diseases, and genetic disorders that were once untreatable, making them powerful therapeutics. Research into the methodology of gene therapies began in 1987 (NIH) when scientists came across an intriguing repetitive DNA sequence as they analyzed genes related to phosphate metabolism within the Escherichia coli genome. This sequence was later identified as a CRISPR.

​​Jennifer Doudna and Emmanuelle Charpentier revolutionized gene therapy by developing the CRISPR/Cas9 gene editing technique in 2012. They’ve been awarded the Nobel Prize for this discovery in 2020. This technology enabled scientists to edit the human genome, targeting disease-causing errors at their root cause, DNA. Eleven years later, the FDA approved a record-breaking five gene therapies, including the first CRISPR-based treatment for sickle cell disease, Casgevy (by Vertex Pharmaceuticals and CRISPR Therapeutics), changing the lives of patients living with this terrible disease. (Nature)

However, getting to this point has not been easy. Each step of the process has its own challenges that need to be resolved, including science, manufacturing, regulatory processes, and communication between companies, investors, and patients.

With more gene therapies being approved, we know that they have the potential to be the solution for many diseases. However, precise delivery of the genetic material still poses a great challenge — how to ensure delivery to the correct location and how to transport a heavier genetic load? Taking this to a larger scale, the manufacturing process must be reproducible and cost-effective, which requires a highly skilled workforce.

Building a regulatory framework around a novel therapy is a slow process, and gene therapy is no different. Safety procedures and efficacy requirements need to be considered, which puts a lot of pressure on regulatory institutions like the FDA. There is a delicate balance between doing it right and doing it quickly so they can simultaneously ensure patient safety and not delay treatment to those who need it the most.

In light of the industry’s greater challenges, communication seems to be the least of its problems, even though it is an ongoing need. Specifically, how can one-time treatments be made attractive to investors and justify the evaluation based on this metric? Or the challenge of communicating effectively with patients, which often leads to slower recruitment times for clinical trials and creates significant delays in the development of new therapies.

All gene therapy companies encounter these difficulties. We work with clients in this field and observe firsthand how they pave the way for the industry to move forward with their innovative science while resolving day-to-day challenges.

For example, scientists are exploring non-viral vectors that have the potential to be safer, more cost-effective, and capable of delivering larger loads of genetic material. (ASGCT). Our client, ReCode Therapeutics, is at the forefront of developing such technology – a novel lipid nanoparticle (LNP) delivery mechanism that enables gene therapies to be delivered to the targeted organ. ReCode has a pipeline of therapies targeting rare diseases and is currently testing its first program in clinical trials. As a marketing and communications agency, we support them in their demanding journey, finding creative solutions to help them with daily needs and communicating with investors and interested parties.

Another client of ours that has been creatively resolving common obstacles in this industry is Lexeo Therapeutics – a public company targeting genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease. We’ve been supporting Lexeo in its journey from private fundraising to its IPO, finding creative solutions to their investor communication challenges. As they approached clinical trials, they encountered another obstacle – how to explain gene therapies to patients. We’ve been helping the Lexeo team develop patient materials by combining simple illustrations with patient-friendly language to explain gene therapy science and Lexeo’s programs.

Currently, there are 2111 gene therapies in clinical development, with oncology and rare diseases being the most common therapeutic areas (ASGCT). The FDA has approved a total of twelve gene therapies to date, with more approvals expected in 2024. The increasing popularity of gene therapy has created more investment opportunities as gene therapy companies enter the public market through initial public offerings (IPOs). Earlier this year, eight biotechnology companies went public, raising $1.2 billion combined. (Biopharmadive)

The gene therapy market is fast-growing and continuously evolving. The human genome offers a vast array of targets, providing endless possibilities for innovative therapies. We look forward to seeing advancements in this field and future FDA approvals that will make a difference in patients’ lives.

About Theoria Creative

We specialize in bridging the communication gap for our clients by helping them effectively convey their distinct messages and value to investors. However, we also support our clients in developing their patient-facing communications. We can distill your complex science into simple, patient-friendly language with engaging visuals. With our expertise in both science and marketing, we are well-equipped to handle your investor and patient materials.