Gene therapy: A promising future at a high price

Gene therapy harnesses the body’s capacity to heal itself

April 10, 2020

Gene therapy modifies a person’s genes to treat or cure disease.1 Unlike most medications, which work to address symptoms or slow progression of an illness, gene therapy aims to correct an underlying genetic disorder. Gene therapy can work in several different ways:

  • Replacing a disease-causing gene with a healthy copy of the gene
  • Inactivating a disease-causing gene that is not functioning properly
  • Introducing a new or modified gene into the body to help treat a disease1

The idea for gene therapy was first published in 1972, but practical applications weren’t approved in the United States until 2017.

What are the applications for gene therapy?

Gene therapy can be applied in a number of areas including:

  • Oncology
  • Musculoskeletal
  • Central nervous system
  • Endocrine, metabolic and genetic disorders
  • Cardiovascular
  • Hematology
  • Ophthalmology
  • Immunology and inflammation
  • Infectious diseases
  • Dermatology
  • Gastroenterology
  • Respiratory
  • Geriatrics
  • Lymphatic, and more

Gene therapies do not always completely cure a disease, but in many cases may improve patient outcomes. Although gene therapy demonstrates a promising future, we are still learning a lot about it and its durability (how long its effects will last in the body).

Gene therapy has a strong outlook

The U.S. Food and Drug Administration (FDA) anticipates that each year they will receive more than 200 investigational new drug (IND) applications for new gene therapy. By 2025, the FDA expects to approve 10 to 20 new gene and cell therapies per year.2

Total of 352 clinical trials underway in Q4 2019

Gene Clinical trials underway in Q4 2019

Clinical data numbers for chart were referenced from Alliance for Regenerative Medicine3

Gene therapy costs are high

Gene therapy comes with a high cost, due to its complexity to produce and small population to treat.4 The FDA has approved two gene therapies.

Luxturna®: In December 2017, the FDA approved Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl)5 for the treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy. It is administered as two separate subretinal injections, one per eye. The wholesale acquisition cost (WAC) of Luxturna is $850,000 per person or $425,000 per eye, not including medical costs.

Zolgensma®: AveXis’ Zolgensma® (onasemnogene abeparvovec-xioi), is FDA approved for the treatment of spinal muscular atrophy (SMA) in patients under the age of 2 years old. The WAC for a one-time intravenous infusion of Zolgensma is $2.125 million per treated patient.6

FDA-approved gene therapy products

FDA-approved gene therapy productsPreserveRxSM will help support Blue Plans

The number of gene therapies for rare conditions are growing. So are the costs. An estimated 60 gene therapies will come to market in the next 10 years. Prime offers financial protection for health plans that helps preserve patient access to innovative gene therapies.2

Prime, together with BCS Insurance Company, has announced a robust gene therapy reinsurance product called PreserveRxSM PreserveRx seeks to protect both health plans and employers from sudden, one-time costs due to covering ultra-expensive gene therapies, while still preserving access to these potentially life-changing or curative gene therapies for members.

The initial PreserveRx offering will help provide financial protection to Blue Cross and/or Blue Shield Plans for five high-cost gene therapies: Luxturna® and Zolgensma®, plus three additional gene therapies that may be approved by the FDA this year: valrox, LentiGlobin®, and GT-AADC.

Prime keeps clients ahead of the drug pipeline

Prime posts new drug approvals in its monthly pipeline updates. Prime also posts pipeline projections quarterly, in its March, June, September and December specialty pipeline updates.


  1. “What is Gene Therapy?” U.S. Food & Drug Administration. Accessed 4/7/20:
  2. FDA Statement from Commissioner of Food and Drug Administration Scott Gottlieb M.D. on Jan. 19, 2019. Accessed 4/7/20 at:
  3. “Advancing Gene, Cell, & Tissue-Based Therapies.” Alliance for Regenerative Medicine 2019 Annual Report. Accessed April 7, 2020.
  4. “Cell and Gene Therapies Face Manufacturing Challenges,” Jan 01, 2017. By Cynthia A. Challener. BioPharm International. Volume 30, Issue 1, pg 20–25. Accessed 4/7/20 at:
  5. FDA Approval Letter. Accessed 4/7/20 at:
  6. “At Over $2 Million Zolgensma Is The World’s Most Expensive Therapy, Yet Relatively Cost-Effective,” by Joshua Cohen. Forbes. June 5, 2019. Accessed at:

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