The Coming of Age of Gene Therapy


In vivo gene therapy, the process of delivering genetic material into the body to cure disease, is a challenging area but has the potential to cure millions suffering from genetic conditions such as cystic fibrosis or muscular dystrophy. With a $40b plus market, gene therapy has the potential to be non-pathogenic to humans and prevent DNA damage and unpredictable consequences. While there have been many failures, gene therapy is now hot, with M&A activity following suit. Biopharmas are now racing to buy validated gene therapy delivery platforms or be shut out from the space.

In 2018, Novartis paid $8.7b for AveXis Therapeutics, a developer of therapies for neurological genetic diseases. In May 2019, AveXis won approval for its spinal muscular atrophy gene therapy Zolgensma.

• Catalent, Inc. (NYSE: CTLT) -- with revenues of $2.5b and a market cap of $7.25b -- has been extremely active on the M&A front.

In 2018 it bought UK-based Juniper Pharmaceuticals, an expert in early-phase product development for $133m.

Also in 2018, Catalent paid $950m for Cook Pharmica, a biologics-focused contract devel-opment and manufacturing organization (CDMO) with capabilities across biologics development, clinical and commercial cell culture manufacturing, formulation, finished-dose manufacturing, and packaging. Cook Pharmica’s revenue is about $179m.

In April 2019, Catalent bought Paragon Bioservices, a developer of viral vectors for gene therapies, for $1.2b. Paragon engages in the production and purification of therapeutic proteins, monoclonal antibodies, viral vectors, and vaccines-VLPs.

In March 2019, Thermo Fisher Scientific paid $1.7b for Brammer Bio, a CDMO focused on manufacturing viral vectors for gene and cell therapies. Brammer offers pre-clinical and analytical development, process optimization, analytical qualification as well as clinical and commercial drug substances. Brammer Bio’s revenue is about $250m.

Also in 2019, Biogen paid $800m for UK-based Nightstar Therapeutics, a clinical-stage gene therapy company focused on adeno-associated virus treatments for inherited retinal disorders.

• Roche is seeking to buy Spark Therapeutics for $4.8b. Spark has an approved gene therapy treatment, Luxturna, as a potential one-time treatment for a rare, genetic form of blindness. Spark also has a gene therapy program for hemophilia. The deal is currently under antitrust review.


Keywords: Healthcare Investment Banking, Healthcare M&A, Healthcare mergers