Genome therapy is one of the fields in medical sciences that is growing rapidly. This therapy is aimed at treating inherited or genetic diseases by substituting or repairing the defective genes.

This particular biotech industry division comprises of a number of small and big firms and presents numerous opportunities for investment in genome therapy. These investments opportunities are best-suited for companies in three categories, including:

1. Companies that are potential targets for M&A (mergers and acquisition)
2. Companies that are strong and lucrative standalone units
3. Corporations that are flourishing through M&A

Top Players with Notable Investment in Genome Therapy

While there are hundreds of small and big investors in this area, below is the list of top ten players with remarkable investment in genome therapy, as endorsed by Barron’s.

• Spark Therapeutics Inc. (ONCE), $4.3 billion
• Sarepta Therapeutics Inc. (SRPT), $9.7 billion
• Regenxbio Inc. (RGNX), $2.0 billion
• Voyager Therapeutics Inc. (VYGR), $0.6 billion
• MeiraGTx Holdings PLC (MGTX), $0.5 billion
• Audentes Therapeutics Inc. (BOLD), $1.5 billion
• Novartis AG (NVS), $227.6 billion
• Roche Holding AG (RHHBY), $230.9 billion
• uniQure NV (QURE), $1.9 billion
• Solid Biosciences Inc. (SLDB), $0.4 billion

Significance for Investors

Research shows that a single flaw in genes or a mutation can lead to as many as 5,000 unusual diseases. But the good news is that these can be cured with corrective genome therapy.

While these diseases are rare and affect a limited number of people, successful treatment for these can easily produce revenue of over $1 billion annually. This is mainly because such procedures are expensive.

Nonetheless, even after being backed by decades of research and experimentation, the concept of gene therapy is still in the developmental stage. It is evolving and improving every year, and has a wide scope due to commercialization. This is what has made investments in genome therapy a worthwhile opportunity.

Thus far, the only type of gene therapy that has been approved by the FDA is one that was introduced by Spark Therapeutics in 2018 to fix an unusual eye disorder that ultimately causes blindness. In its debut year only, the development was able to generate revenue of $27 million.

The Scope of Investments in Genome Therapy

While the commercialization of genome therapies is occurring at turtle’s pace, the future of investors in this area looks bright. The reason behind slow-paced commercialization is that there are several small players in the field holding large R&D stores but, of course, with a limited capability to bear costs.