A black-and-white photo of a young girl with curly hair looking directly into the camera. She is wearing a t-shirt and appears to be indoors, with a blurred background of furniture and a computer monitor.

We are engineering a new model to make and commercialize medicines
for patients with genetic diseases. At scale.

A woman lifts a smiling little girl with curly hair into the air outdoors, with trees and a fence in the background.

We are a team of scientists, biotech professionals, and parents making medicines for suffering and dying children with genetic diseases.

Most of the 200 million children with a rare genetic disease will never see a treatment due to the current healthcare model.

We have discovered genetic technologies that can treat many of these diseases, and developed a business model that will bring treatments to many of these children. 

AlphaRose Therapeutics is a new precision medicine model for genetic treatments.

We stand on the foundations laid by pioneers such as Henri Termeer and the resilience of patient families.

We are born from the incredible stories of precision medicine for children, like that of Rose McPherson.

We exist to transform the delivery of genetic treatments for patients, advancing them faster and on a larger scale, beginning with the children who need it most.

Rare disease is not rare. Treatments are.

250+

new rare diseases discovered every year.

10,000

rare genetic diseases.

Less than 50

new rare treatments are approved every year.

200 million

children suffer from them.

5%

have treatments.

30%

of those children die before their 5th birthday.

This is unacceptable.

A young girl with glasses smiling and raising her arms, cluttered room background.
A crowded city street with people, some wearing winter clothing, and a child sitting on someone's shoulders in the foreground. Tall buildings and a bright sky are visible in the background.

We have the technology to cure many of these diseases, now we must build the systems to do it at scale. 

Patients are waiting.