re: Generation

June 22, 2020

Penny and Simon take on the world

Simon and Penny jumping

Penny and Simon aren’t your typical brother and sister.

Born from parents Danny and Jaime, who share a profound sense of wanderlust, they are already globe-hopping travelers, who have explored 12th century churches and uncovered mysteries in the rainforest. They traced the course of their ancestors across continents. In Cuba, they experienced what life was like under rationing — unable to buy milk or bread. They’ve explored Croatia, Bosnia and Herzegovina, Montenegro, Italy, and the Canary Islands.

They were also born with glycogen storage disease 1a (GSD1a), a rare genetic condition which inhibits their ability to process sugars. So, between building forts and climbing mountains, Penny and Simon have to have their blood sugar levels checked at least seven times a day. And they have to meticulously nourish their bodies every three hours, around the clock, with a starchy substance taken in through a gastronomy tube (G-tube), until they are old enough for their bodies to handle a cornstarch slurry they can drink out of a glass.

"There is no normal. There is only life." — Danny

Penny with her cornstarch slurry

Penny is now able to tolerate drinking a cornstarch slurry, which means she can now go three to four hours between feeding during the day. She is still fed through her G-tube at night.

With such challenging implications of GSD 1a, many in their family have questioned Danny and Jaime’s choices to eschew the safety of a home and salaried jobs. However, they have income through internet-based work and a steadfast commitment to trading fear for decisions based on love — for each other, for exploration and the strangeness of the world, for the inevitable difficulties of living this way.

"I enjoy seeing different, new places, and exposing the kids to that. Showing them that there are a lot of different people in the world, but we're all more-or-less the same. We all have the same needs." — Jaime

Yes, this life comes with its challenges. A simple stomach bug can become a life-threatening situation. And their parents have had to become seasoned experts, often informing the doctors who are informing the medical decisions. They have to enter emergency departments armed with histories, medicines, and protocols that are critical to their children’s health.

Having a rare disease could conjure up images of the unspeakable, but Danny and Jaime refuted the fearful inclinations. Instead, having GSD1a is all the more reason to get on a plane, a boat, or a bus, and show their kids the world. In stride, they have found a measure of balance between living on the road and living with genetic disease. It has just meant they have had to meticulously pack a few more suitcases.

"We haven't created a negative connotation for GSD with them. We haven't made it a heavy thing. We made it like: This is our lives. This is what you have. This is how we keep you healthy. We can still do normal things. We can still do what other people do. It sometimes might be harder. It might be more work. We have to take precautions." — Jaime

Our platform currently targets three diseases of the liver. It’s our mission to expand our portfolio to include GSD1 and many more—to unlock the potential of gene therapy for families everywhere.

Simon and Penny playing on the slide

Every day we come to work focused on Penny and Simon, and a day when families like theirs can move through this world having no understanding of how genetic disease would have once affected their lives. When they can leave those extra suitcases behind and focus solely on the world that lies ahead. Because kids should be free to be just kids. And parents should be free to be just parents. And life should be free to be one non-stop adventure.

Safe Harbor Statement: Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, the expected timing of the submission of IND applications and preclinical data, our manufacturing plans, our expectations regarding our new facility and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the company’s product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties regarding the timing and ability to complete the build-out of the Company’s manufacturing facility and regarding the new manufacturing process; expectations regarding the timing of submission of IND applications; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the Company’s cash resources are sufficient to fund the company’s operating expenses and capital expenditure requirements for the period anticipated, including the funding of the new manufacturing facility; the impact of the COVID-19 pandemic on the company’s business and operations; as well as the other risks and uncertainties set forth in the “Risk Factors” section of our most recent annual report on Form 10-K and quarterly report on Form 10-Q, which are on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof. The company anticipates that subsequent events and developments will cause the company’s views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date on which they were made.

Take a seat at the table.

We are always looking for those who are ready to share their vision, talent, and tenacity—who believe in our mission and themselves.

Imagine a world without genetic disease.

What do you see? More health? Fewer doctor appointments? More bike riding? More peanut butter and jelly sandwiches? More life?
See our shared vision.