Gene Therapy Market: Competitive Landscape, Pipeline, and Market Analysis 2024

Genes are segments of DNA that contain the instructions for building and maintaining the structures and functions of living organisms. Each gene encodes specific information in the form of nucleotide sequences, which are the building blocks of DNA. These sequences provide instructions for the production of proteins or functional RNA molecules, which are essential for carrying out various cellular functions. Gene therapy is a medical approach that involves manipulating the genetic material of a person's cells to treat or prevent diseases. The main goal of gene therapy is to correct or replace faulty genes, introduce therapeutic genes, or regulate the expression of genes to alleviate the underlying cause of a genetic disorder or disease.

Gene therapy involves the partial or full replacement of defective genes in a patient's body with healthy ones to treat or prevent disease proliferation. There are two types of gene therapy based on the location of the target gene on a chromosome including somatic gene therapy & germline gene therapy. It can be applied for the treatment of several diseases, such as Parkinson’s, hemophilia, cystic fibrosis, Alzheimer’s, brain tumors, cancer, AIDS, SCID, and others. The transgene is transferred into the host cell by biological vectors, such as the virus, liposome, and plasmids. These vectors are further categorized as viral vectors & non-viral vectors. The viral vector consists of retrovirus, lentivirus, adenovirus, and adeno-associated vector. The non-viral vector includes liposomes, transposons, and plasmids. Moreover,  an increase in cases of genetic disorders and CNS disorders are the key factors for driving the growth of the Gene Therapy market. For instance, according to the Centers for Disease Control and Prevention (CDC), Parkinson's disease is estimated to affect around 1 million people in the United States in 2021. The introduction of newer drug products by many market players may aim to influence significant research and development in Gene Therapy. For instance, Enzyvant launched Rethymic to treat congenital athymia. Moreover, to overcome challenges in therapy, various market players are developing innovative compounds in the various stages of clinical trials.

Key Market Developments:

• In October 2021, Enzyvant received FDA approval for its Rethymic (Allogeneic Processed Thymus Tissue–Agdc) indicated for immune reconstitution in pediatric patients with congenital athymia.
• In June 2023, BioMarin received FDA approval for its Roctavian (Valoctocogene Roxaparvovec-Rvox) indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5.

Approved Drug Molecules and Brand Names for Gene Therapy:

• Roctavian (Valoctocogene Roxaparvovec-Rvox)
• Rethymic (Allogeneic Processed Thymus Tissue–Agdc)
• Elevidys (Delandistrogene Moxeparvovec-Rokl)
• Zolgensma (Onasemnogene Abeparvovec-Xioi)

Clinical Activity and Developments of Gene Therapy :

Till July 2023, more than x companies have approximately x molecules targetting Gene Therapy. For these molecules, more than x clinical trials are being conducted; most are in phase-1 and phase-2 clinical trials by players across the globe. For instance,

• In May 2019, Novartis received FDA approval for its Zolgensma (Onasemnogene Abeparvovec) based on the STRIVE, an ongoing, open-label, single-arm clinical trial, that evaluated Zolgensma in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene.