Rare Disease Report

Pfizer buys gene therapy company - Is it a good investment?

AUGUST 01, 2016
James Radke, PhD
Pfizer has acquired gene therapy company Bamboo Therapeutics for a deal that could be worth up to $645 million.
Bamboo therapeutics is developing gene therapy for Giant Axonal Neuropathy (GAN) that is currently in phase 1/2 trial. The company also has gene therapies in development for 3 other neuromuscular diseases – Duchenne muscular dystrophy, Canavan, and Friedreich’s ataxia.
One group that is particularly excited about this new opportunity is CureDuchenne.  Debra Miller, CEO of CureDuchenne Ventures and CureDuchenne said, “Bamboo Therapeutics is delivering some of the most promising work in gene therapy. Their progress is giving hope to this and future generations of boys with Duchenne and their families.”
“Bamboo’s transformational approach is leading the way to new medical breakthroughs where genetic mutations will one day be corrected

Rare neurological and neuromuscular diseases targeted

Giant axonal neuropathy (GAN) is caused by mutations in the GAN gene, which codes for the protein gigaxonin that usually forms neurofilaments. In the absence of functional gigaxonin, other proteins are toxically communicating, causing densely packed, swollen and giant axons, which interferes with the signaling between the peripheral nervous system and the central nervous system. Most children become wheelchair dependent in the second decade of life. Some children may survive into early adulthood.
Duchenne muscular dystrophy (DMD) is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10.
Canavan is a progressive neurological genetic disorder caused by the absence of aspartoacylase (ASPA) which breaks N-acetylaspartate acid (NAA) into building blocks essential for building myelin. Death usually occurs before age 10 but some children with milder forms of the disease survive into their teens and twenties.
Friedreich’s ataxia (FA) is caused by mutation of the FXN gene that leads to low levels of frataxin that is involved in mitochondrial functions. Disease severity and progression vary but symptoms often appear between the ages of 5 to 18. Most young people diagnosed with FA require mobility aids such as a cane, walker, or wheelchair by their teens or early 20s.

Big Money

The buyout gives Bamboo shareholders $150 million up front and they could receive an additional $495 million depending on milestones. This acquisition provides Pfizer with an advanced recombinant Adeno-Associated Virus (rAAV) vector design and production technology, and a fully functional Phase I/II gene therapy manufacturing facility that Bamboo acquired from the University of North Carolina earlier this year. Bamboo has fewer than 40 employees. Most have been offered a position with Pfizer.

Does gene therapy work?

In a recent editorial in JAMA, Drs Micheal Joyner, Nigel Paneth, and John Iannidis, of the Mayo Clinic, Michigan State University, and Stanford University, respectively, noted that the moonshot idea of gene therapy to cure patients has long been studied but results are extremely limited.
The doctors noted that in 2016 approximately $15 billion of the $26 billion of extramural research funding sponsored by the NIH could be linked to some version of search terms that include gene, genome, stem cells, or regenerative medicine. And the doctors used the example of sickle cell anemia to illustrate the difficulty in developing a gene therapy.
Joyner et al stated, “Continued enthusiasm for gene therapy ignores what is known from classic single-gene disorders such as sickle cell anemia. The complex biological processes set in motion by a single amino acid substitution that leads to painful crises, stroke, and other complications are not predictable from the genomic defect, but only by appreciating the complexity of biological systems at the level of tissues and organs. Sixty years after the discovery of the genetic defect, no targeted therapy has emerged for sickle cell anemia.”

To date, only one gene therapy has ever been approved. And it was approved only in Europe and it is unclear how financially successful it was. With that being said, there are a LOT of companies currently looking at gene therapy for a variety of medical conditions.

I am cautiously optimistic that gene therapy is the future of many rare disease conditions but it is a lot harder than many believe and it is still unclear how it can be profitable in the end. What can a company charge for a single gene therapy infusion that cures a patient and still make a profit?

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