Investing that Makes the World Rejoice: Gene Therapy
Article originally posted here by Eventide Asset Management
Eventide was founded with a vision of “investing that makes the world rejoice.”
Nowhere is this vision of investing more clearly seen than in biotechnology, where human creativity is being channeled to eradicate and treat devastating diseases. Here we find investment opportunities that can result in hope for patients and families.
One such story is for a little girl named Evelyn Villarreal, and for her parents, Milan and Elena.
Evelyn was diagnosed at birth with a rare genetic disease called spinal muscular atrophy type 1 (SMA1). As the name implies, it is a disease that causes the muscles to atrophy. Babies born with SMA1 have little muscle function; they are limp and floppy at birth, and have difficulty nursing and swallowing.
The disease is caused by a genetic mutation where motor neurons cannot make a necessary protein called SMN (survival motor neuron). And without the ability to make this protein, motor neurons die leading muscles to progressively atrophy to the point where even breathing becomes impossible. The mortality rate is nearly 80% by 24 months and no children with SMA1 are ever able to sit, stand, or walk.
For the parents Milan and Elena, the news that Evelyn had SMA1 was devastating. Unimaginably, they had already lost one child to SMA1, their daughter Josephine, who died at 15 months of age.
Ten years ago, if you were a child born with SMA1, your chance of making it to adulthood was almost 0%. Today, children with SMA1 have a future. How? Biotechnology companies are boldly addressing SMA1 through new treatments such as gene therapy. And investors are capitalizing this important work.
Gene therapy is a targeted treatment that delivers a healthy copy of the right gene to the right cells, promoting normal cellular function. The SMA1 gene therapy uses a virus to deliver a healthy copy of the SMN1 gene to motor neurons so they can produce the protein SMN, thereby enabling normal survival, development, and function.
Evelyn received a gene therapy for SMA1. Here she is, now three years old, doing push-ups!
In fact, 100%(!) of the children who received the full dose of the SMA1 gene therapy were alive at 20 months.
We live in the most exciting time in the history of medicine. Advances in biotechnology have now made it possible to meaningfully fight diseases that previously were a death sentence. Through next-generation gene sequencing and targeted gene therapy, scientists have been able to discover, and doctors now treat, the root causes of diseases like SMA1.
Investors in Eventide are doing more than simply investing for their future – as investors, they are partnering with companies that are replacing sorrow with rejoicing in the lives of those like Evelyn, Milan, and Elena. We invite you to join us in this mission.
Learn more about Spinal Muscular Atrophy (SMA) at http://www.curesma.org/