As we close the year, we were happy to have so many people join us for our much-anticipated cell and gene pipeline webinar.
“Insights & Analysis 2024 Preview: Cell & Gene Therapies in the Drug Pipeline” was co-presented by Two Labs and Envision Pharma Group and featured expertise from our VP of Commercialization Strategy Kimberly Cline and our Drug Pipeline Manager Ryan Chandanais. Together, they highlighted critical insights into cell and gene therapy advancements, focusing on the strategic implications for the near future.
What makes cell & gene therapies so unique?
Primarily falling into the category of regenerative medicine – cell therapies aimed at restoring tissue and organ function and gene therapies that modify genetic material to address diseases – cell and gene therapies bring many unique considerations for how they are carried through the launch process and delivered to patients.
Types of Cell and Gene Therapies
One-time dosing, a characteristic of many cell and gene therapies, poses distinctive challenges, particularly when managing adverse reactions. Unlike traditional drugs administered over several doses, these therapies often do not offer the option to adjust doses post-administration, underscoring the FDA’s cautious approach towards them.
The high cost of these therapies is an obvious barrier. In fact, cell and gene therapies take many places on the list of the 10 most expensive drugs available. But, innovative pricing solutions, such as value-based care, are on the horizon. A critical question of how to pay for the medicines hinges on another essential question: durability. Are the therapies truly curative, or does efficacy wane over time?
What’s in the pipeline?
Turning to the pipeline overview, we find that more than 4,000 cell and gene therapies are in development, indicating a surge in interest and investment in this field. The landscape is rich and diverse, from T-cell therapies tackling cancer in novel ways to stem cell treatments with the potential to replace diseased cells.
The recent approval of CRISPR-based treatments for sickle cell disease and the promising pipeline for conditions like melanoma and hemophilia showcased a future where once-intractable diseases could be managed or even cured.
Where do we go from here?
The pressing question on many minds is how to expand these innovative therapies to larger patient populations and less severe disease states. While cost remains a significant barrier, the life-changing potential of cell and gene therapies ensures the industry’s commitment to overcoming it.
As it has been for many years now, it’s clear that cell and gene therapies will play a critical role in shaping the future of medicine.