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Next in gene therapy: 2024 roundup

What payors can expect for top therapeutic areas


From the Editors


Projected Treatments and Approval Timelines

Gene Therapy Pipeline | Q4 2023 – Q4 2027

Download the report (PDF)


The pipeline for gene therapies is strong and growing. There are 12 products on track for approval in the coming year, according to our latest quarterly update.


More than 20 new gene therapy approvals anticipated through 20251


Among the many therapies expected to launch in 2024, some specialty conditions to keep an eye on include:

Sickle cell disease – Gene therapy options for treating sickle cell disease may soon go from zero to two.

Chronic lymphocytic leukemia – We soon may see the first CAR T-cell therapy for this blood cancer that affects a large patient population.

Beta thalassemia – An alternative treatment option may be available for this inherited blood disorder that currently requires frequent, lifelong blood transfusions.

Hemophilia B – A new alternative to existing treatments may provide long-lasting protection from bleeding.

As plans prepare for the impact of gene therapies launching over the next five years, it’s important to pay attention to key therapeutic areas in the pipeline. Top categories for new gene therapy products (without supplemental indications):

  • Metabolic disorders: 7 products
  • Hematology: 6 products
  • Hematology–oncology: 4 products
  • Ophthalmology: 4 products

This field continues to expand fast. That’s why we closely watch the pipeline to help you better understand the benefits and financial risks of these cutting-edge therapies. Our solutions can help balance equitable coverage and affordability.


  • 1 RxPipeline, November 2023.


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