Gene therapy, a ground breaking technique that alters a person’s genetic material to combat various diseases, is gaining immense traction in the global healthcare landscape. As the prevalence of cancer continues to surge worldwide, gene therapy holds the promise of not only treating cancer but also addressing a wide range of genetic disorders and chronic ailments. The growing investments in gene therapy research further solidify its potential for significant growth in the global market.

The Power of Gene Therapy
Gene therapy encompasses various mechanisms to combat diseases:

1. Gene Augmentation: Introducing new genes to help fight diseases.
2. Gene Inhibition: Replacing mutated disease-causing genes with healthy copies.
3. Targeted Cell Inactivation: Disabling malfunctioning genes.

This transformative approach is classified into two categories:

• Somatic Gene Therapy: Modifying genes in non-reproductive cells, ensuring effects do not pass to future generations.
• Germline Gene Therapy: Altering genes in reproductive cells, potentially impacting future generations.

One of the most widely used techniques employs recombinant DNA (rDNA) technology, where genes of interest are inserted into vectors, such as viruses, plasmids, or nanostructured vehicles, to deliver therapeutic genes efficiently.

Diverse Gene Therapy Products
Gene therapy products are diverse and include:

• Plasmid DNA: Circular DNA molecules engineered to carry therapeutic genes into human cells.
• Viral Vectors: Modified viruses used as vehicles to deliver therapeutic genes safely.
• Bacterial Vectors: Bacteria modified to transport therapeutic genes.
• Human Gene Editing Technology: Focusing on disrupting harmful genes or repairing mutations.
• Patient-Derived Cellular Gene Therapy Products: Cells removed from patients, genetically modified, and reintroduced to treat specific conditions.

In this process, target cells, such as those in the liver or lungs, are infected with the vector, which then delivers therapeutic genes. This leads to the production of functional proteins, restoring the target cells to a normal state.

Challenges in Gene Therapy
While gene therapy offers great promise, it faces several challenges:

• Safety Concerns: Viruses used in gene therapy are modified for safety, but concerns linger.
• Long-Term Effects: It is uncertain how long the effects of gene therapy will last.
• High Costs: Gene therapies are among the most expensive treatments, often customized for each patient.

For instance, Kymriah, used for pediatric leukemia and advanced lymphoma, can cost up to US$475,000 in the US. Luxturna, prescribed for inherited retinal dystrophy, carries a price tag of US$850,000, while Zolgensma, a life-saving treatment for pediatric spinal muscular atrophy, costs an astounding US$1.5 million per patient.

A Lucrative Frontier in Biotechnology
Gene therapy stands as one of the most remarkable areas of biotechnology today, with unprecedented success in treating diseases once deemed untreatable. With nearly 80% of rare diseases having an identified genetic origin, gene therapy offers hope to millions worldwide.
Rare diseases affect approximately 30 million Americans and 400 million people globally, often impacting more individuals than cancer and AIDS combined. The quest to unlock the potential of gene therapy has spurred established and emerging companies to develop pioneering breakthroughs, potentially changing patients’ lives.
The gene therapy landscape is evolving rapidly, with over 400 gene therapy trials underway. In the US alone, there were 854 companies developing such therapies when the first gene therapy was approved. By the end of 2020, this number had surged to 1,085 companies.

A Highly Regulated Field with Vast Potential
As investigational gene therapies continue to demonstrate clinical and commercial potential, both small and large biotech players are positioning themselves to harness these opportunities. Recent notable acquisitions, such as Bayer’s purchase of AskBio Pharmaceuticals for $4 billion and Eli Lilly’s acquisition of Prevail Therapeutics for $1.5 billion, highlight the industry’s momentum.
Gene therapy is subject to stringent regulation by bodies like the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) and the National Institutes of Health (NIH). However, despite this regulation, approximately 95% of rare diseases still lack FDA-approved treatments. The European Union has its own regulatory framework through EudraLex and the European Medicines Agency (EMA).

Leading Players in the Gene Therapy Market
Key players driving advancements in the global gene therapy market include Asklepios BioPharmaceutical, CRISPR Therapeutics, Editas Medicine, Homology Medicines, Intellia Therapeutics, Passage Bio, Pfizer, Poseida Therapeutics, Prevail Therapeutics, Roche, Sangamo Therapeutics, Bluebird Bio, Kura Oncology, Translate Bio, Krystal Biotech, Replimune, StrideBio, Cardior, Generation Bio, Evox Therapeutics, and Mediphage Bioceuticals.

Global Industry Analysis (2018 – 2021) – Growth Trends and Market Forecast (2022 – 2029)
https://www.fairfieldmarketresearch.com/report/gene-therapy-market

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