An Overview of Cell and Gene Therapies

Since 2017, four new cell and gene therapies have become available in the U.S. with prices ranging from $373,000 to $2.1 million for a one-time administration.

Gene Therapy Launch Timeline

Gene Therapy Product Overview

Product Indication Therapy Type Price
Kymriah
  • Childhood B-cell acute lymphoblastic leukemia (ALL)
  • Diffuse large B-cell lymphoma (DLBCL)
CAR-T cell therapy $475,000 / treatment
Yescarta
  • Diffuse large B-cell lymphoma (DLBCL)
CAR-T cell therapy $373,000 / treatment
Luxturna
  • Biallelic RPE65 mutation-associated retinal dystrophy
Gene therapy $425,000 / eye
Zolgensma
  • Spinal muscular atrophy (SMA)
Gene therapy $2.1 million / treatment

There are an additional 17 approved cell and gene therapies and the FDA anticipates that by 2025 they’ll be approving between 10-20 cell and gene therapies a year.

For the biopharma industry, there is an opportunity to develop life saving treatments. For example, inherited retinal insufficiency and inherited blindness can be treated, blood disorders such as hemophilia and sickle cell disease can be treated, and even cancer can be cured in some cases.

Cell and gene therapies are capable of incredible things, but they are not without challenges. CAR-T sales have been lower than anticipated. Zolgensma sales have been promising, still only 200 patients were treated in 2019.

It is important for manufacturers to partner with the right teams to plan out the patient journey, craft distribution strategies, and properly time customer onboarding and launching in order to achieve commercial success.

Two Labs can partner with clients to help navigate cell and gene therapy launch

A few of our capabilities include:

  • New product launch planning
  • Creation of distribution strategy and selection of distribution vendors
  • Development of patient journey paths and selection of patient services vendor
  • Coordination of meetings and conferences with the commercialization vendors
  • Market and competitive landscape assessment and planning
  • Pricing and market access strategy development

Our team of experts are uniquely positioned to assist cell and gene manufactures with a full deck of commercialization services. With extensive biopharma industry experience and a deep bench of subject matter experts, we’re able to assist cell and gene manufacturers every step of the way.

To learn more about how Two Labs can help you from commercialization strategy to implementation, please visit TwoLabs.com/services or contact us at info@twolabs.com 614-389-4000.