ICON_Jan2021_CellandGeneTherapy - 4

The Future is Bright for CGT Therapies

The Future is Bright for
CGT Therapies

T

he promise of cell and gene therapies to

Furthermore, these advances in CGT revolu-

solve some of our most challenging condi-

tionized the field of immunotherapy, particularly

tions, from rare diseases to cancers, has led

oncology. For example, emerging trends in immu-

this field to remain on the forefront of innovation.

no-oncology include chimeric antigen receptor

Advances in innovative technologies coupled with

T-cells (CAR-T), adoptive cell therapy (ACT) and T-cell

deeper scientific knowledge in cellular and molec-

receptor (TCR) therapy. While the majority of invest-

ular biology spurred a new era of growth in these

ment in CGT in oncology focuses on hematologic

advanced therapies. Over the past decade, more than

cancers, more research programmes are shifting to

10 advanced therapy medicinal products (ATMPs),

treat solid tumors as increasing evidence shows the

have received approval in Europe.1 The US is expe-

effectiveness and potential of immunotherapies to

riencing similar growth, with currently 10 approved

treat cancer while achieving prolonged remission.

therapies and the FDA expecting an approval rate of

CGT's impact on immunotherapy has also

10 to 20 cell and gene therapies every year by 2025.2

extended to the COVID-19 pandemic. As reported by

Already, in 2020, we've witnessed continued

the Alliance for Regenerative Medicine, there are 11

growth in approvals, despite the COVID-19

ongoing clinical trials using regenerative medicine

pandemic-from Japan and Europe's approval of

and advanced therapy technologies to treat

Zolgensma (AveXis, a Novartis company),3 a gene

COVID-19, with another 25 programs in preclinical

therapy treatment of spinal muscular atrophy to the

development.6 For example, biotech companies are

US FDA's approval of brexucabtagene autoleucel

using mesenchymal stem cells and other stromal

(formerly KTE-X19; Tecartus from Kite, a Gilead

cells to treat acute respiratory distress syndrome, a

company),4 for the treatment of relapsing or refrac-

serious complication due to coronavirus.

tory mantle cell lymphoma. CGT products account

For the first time, there are more than 1,000

for approximately 12% of the pharmaceutical

active regenerative medicine and advanced therapy

industry's clinical pipeline and at least 16% of its

developers worldwide, and of these companies, 415

preclinical pipeline.5

are in the clinical stage of development.6 As the CGT

4|

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ICON_Jan2021_CellandGeneTherapy

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