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Gene Therapy – Revolutionizing Medicine and a Hope for Millions

June 21, 2023 | Healthcare

Gene therapy is a treatment that includes modification/manipulation of the gene expression with an aim to treat genetic disorders. Gene therapies can be delivered either in vivo or in vitro. The in vivo delivery includes the administration of the viral vector intravenously into the body whereas the in vitro delivery includes the removal of the patient’s cells, administration of the viral vector in those cells, and then the cells containing the vector are reintroduced into the patient.

The growing prevalence of rare disorders with genetic component as a factor among the population globally is one of the major factors contributing to the rising focus of the drug manufacturers to develop and introduce novel gene therapies for the conditions.

  • According to a 2021 article published by the National Human Genome Research Institute (NHGRI), there are more than 6,800 rare diseases and altogether these rare diseases affect approximately 25 to 30 million people in the U.S.

  • Also, according to a 2022 article published by the U.K. government, around 80% of rare diseases have a genetic origin and collectively, rare diseases affect 1 in 17 people at some point of their lifetime.

The rising R&D investment by key players is leading to an increasing number of pipeline products, and the growing number of approvals and introduction of gene therapies by the market players to cater the growing demand are some of the factors contributing to the growth of the market.

  • According to a 2022 report published by the American Society of Gene & Cell Therapy (ASGCT), the pipeline products in gene therapy increased by 6% in 2022 as compared to the previous year.

According to the American Society of Gene & Cell Therapy (ASGCT), as of December 2022, there were more than 2,000 gene therapies in the pipeline globally. The distribution of these therapies on the basis of the clinical phase are mentioned below:

  • Phase I – 1,000+

  • Phase II – 9905

  • Phase III – 120

  • Pre-registration - 25

The below mentioned figure represents the distribution of gene therapies (including genetically modified cell therapies) based on clinical phases, as of 2022.

Gene Therapy – Revolutionizing Medicine

Source: 2022 Report published by The American Society of Gene & Cell Therapy (ASGCT)


Huge Potential Partially Offset by High Costs 

The producing, handling, and controlling the viral vectors are complex as compared to working with chemicals for manufacturing of traditional drugs and therapies. The complex R&D activities result in huge investment costs for the manufacturers. Along with this, gene therapies are mostly for the rare disorders affecting a smaller population, and are given as a one-time treatment to patients. Therefore, these are some of the significant factors responsible for the high cost of the therapies.

The top five expensive FDA approved gene therapies with their respective approval dates are mentioned in the below table:




FDA Approval Date

Cost per Dose (USD Million)



Hemophilia B

November 2022



bluebird bio, Inc.

Cerebral Adrenoleukodystrophy

September 2022



bluebird bio, Inc.

Beta Thalassemia

August 2022



Novartis AG

Spinal Muscular Atrophy (Type I)

May 2019



Spark Therapeutics, Inc.

Biallelic RPE65 Mutation-associated Retinal Dystrophy

December 2017



Pharmaceutical as well as biotechnological companies in the market are continuously focusing on strategic ways to reduce the cost of gene therapies with an aim to increase the accessibility and penetration in the market.

Below mentioned are some of the approaches being adopted by the pharmaceutical companies to overcome the challenge of high cost:

  • Outcome-based Rebates – This approach spread the risk between the payer and the manufacturer. The payer pays the manufacturer the full price of the drug and in the event of drug not meeting the predetermined results, the payer is reimbursed the full cost.

  • Some manufacturers are offering outcome-based contracts that allow health plans to reduce the cost associated with prescription drugs.

    • For instance, bluebird bio Inc., a manufacturer of ZYNTEGLO, announced outcome-based contract offering and a comprehensive patient support program to drive accessibility of the therapy.

  • Subscription Model – As per this model, pharmaceutical companies get an upfront payment from patients who can access the therapies later at comparatively lower prices.

  • Deferred Payments - Some manufacturers provide upfront patient access to the therapies and deferred payments till they complete price negotiations with governments. This allows the reimbursement bodies to manage budget impact during the early access phase.

    • For instance, Novartis received the EC approval and activated the ‘Day One’ access program for Zolgensma.

  • Outcome-based Annuity Payments - This approach allows the manufacturers to spread the cost over years and thereby increasing the eligibility and accessibility of the therapies among the patient population by reducing the overall cost.

  • Outcome-based Installment Payments – This model includes installment payments of the drug spread over a predetermined time period (monthly payments) on the basis of the outcome achieved by the manufacturer that is preset.

Thus, with increasing efforts of manufacturers and other organizations, gene therapies hold a promising potential to deliver lifelong cure for various rare disorders that possess very limited management options. 

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