Patients & families
We are using targeted gene silencing to address neurological diseases at their source
Many CNS diseases are heritable, and genes that contribute to or cause many of these diseases have been identified. Neurodegenerative diseases are incurable, progressive disorders that are rising in prevalence as the population ages.
RNA interference offers a powerful means of silencing disease-causing genes. RNAi blocks the expression of these genes in a way that is controllable and targeted.
Atalanta’s Approach
Many of the most pressing medical challenges in neurology — including Huntington’s disease, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and others — require RNAi to reach deep within the brain.
Atalanta’s proprietary di-siRNA displays unparalleled distribution and durability throughout the central nervous system, including deep brain structures and spinal cord.
About Huntington’s disease
Atalanta’s initial therapeutic focus area is Huntington’s disease, a fatal genetic disorder that causes the progressive breakdown of neurons. Adult-onset Huntington’s disease, the most common form of the disorder, usually appears in a person’s thirties or forties. The disease can cause uncontrolled movements, emotional problems and cognitive decline. Individuals with the adult-onset form of Huntington’s disease usually live about 15 to 20 years after symptoms develop.
Huntington’s disease is caused by mutations in the huntingtin (HTT) gene. The expression of this mutated gene leads to dysfunction in neurons, eventually causing death in certain areas of the brain as the disease progresses.
In animal models of Huntington’s disease, RNAi has shown a promising ability to halt this disease pathology. RNAi can selectively silence the HTT gene using its cellular search function. This prevents the HTT gene from being expressed in the body.
Angela's story
“Music was her life,”
…says her mother, Roz. After Angela was diagnosed with Huntington’s disease at age 33, she was determined to keep dancing.
Angela has a long list of passions. She is a devoted hockey fan (the Chicago Blackhawks are her team), loves dressing up for Halloween, and always wanted to be a contestant on “The Price is Right.”
But her biggest love is music. When she was 16, living in Germany, she played clarinet in an oom-pah band. She excelled at jazz and ballet dancing classes. She went to concerts constantly, or out to karaoke with friends, or to the Eagles Club to dance. Over the decades, as Angela’s life took her to Germany, Holland and then to the U.S., as she was married and raised two children, “music was her life,” says her mother, Roz.
Then, in 2003, Angela was diagnosed with Huntington’s disease at the age of 33. Huntington’s is a neurodegenerative disease caused by mutations in the huntingtin (HTT) gene. The abnormal proteins this gene produces accumulate in brain cells, eventually causing the cells to die. People with Huntington’s therefore have progressive difficulty with voluntary movements, show involuntary movements like jerking or twitching, and experience behavioral and psychiatric symptoms.
The thought that she would someday no longer be able to dance was devastating to Angela.
Angela's story
“Music was her life,”
…says her mother, Roz. After Angela was diagnosed with Huntington’s disease at age 33, she was determined to keep dancing.
Angela has a long list of passions. She is a devoted hockey fan (the Chicago Blackhawks are her team), loves dressing up for Halloween, and always wanted to be a contestant on “The Price is Right.”
But her biggest love is music. When she was 16, living in Germany, she played clarinet in an oom-pah band. She excelled at jazz and ballet dancing classes. She went to concerts constantly, or out to karaoke with friends, or to the Eagles Club to dance. Over the decades, as Angela’s life took her to Germany, Holland and then to the U.S., as she was married and raised two children, “music was her life,” says her mother, Roz.
Then, in 2003, Angela was diagnosed with Huntington’s disease at the age of 33. Huntington’s is a neurodegenerative disease caused by mutations in the huntingtin (HTT) gene. The abnormal proteins this gene produces accumulate in brain cells, eventually causing the cells to die. People with Huntington’s therefore have progressive difficulty with voluntary movements, show involuntary movements like jerking or twitching, and experience behavioral and psychiatric symptoms.
The thought that she would someday no longer be able to dance was devastating to Angela. As her movements became harder to control, she would sometimes fall. Still, she insisted on going out dancing, wearing her most fashionable boots – “no way Angela was going to wear runners,” Roz recalls. And somehow, on the dance floor, Angela always had perfect balance.
“I’d have to stand behind her, I was so scared she was going to fall,” Roz says. But Angela was unafraid of falling or of what others might think. “I’m just doing the new dance!” she’d tell her mom.
“She’s a social butterfly,” Roz says. “We never hid.”
As a young girl growing up in British Columbia, Angela’s favorite TV show was “The Price is Right.” She and her grandmother would tune in every day. She loved the thrill of audience members being selected, the glamorous prizes, and most of all, the charming Bob Barker.
After Angela’s diagnosis, she and her teenage children moved back home to Canada. It was a time of upheaval. Seeking a way to inject some happiness, Roz had an idea. “Going to the Price is Right was the one thing for Angela that was just a dream,” Roz recalls. She started looking into the logistics. And on Christmas 2006, she handed Angela a professionally printed certificate that read “You’re going down to the Price is Right!”
The big day came in 2010. The family, wearing matching shirts that read “Angie’s Dream Team,” took their seats in the studio audience. Each person’s name was written on a big yellow price tag sticker. The studio audience cheered as the camera panned the crowd. Months in advance Roz had written to the show’s producers, asking for the team to get a spot at the podium. The announcer began calling people down. Amidst the frenzy, Angela’s daughter, Ashley, had her name called out. “Angela was crying; we were all crying,” Roz remembers.
After Angela’s diagnosis, the family pursued clinical trials. “Angela wanted to help in any way she could to find a cure,” Roz says. “It was our only link to hope.” She participated in many studies over the years, until the six-hour journey from their home to the University of British Columbia became too hard for her to make.
Alongside her motor difficulties, Angela, like many people with Huntington’s disease, experienced growing irritability and personality changes. She was falling more often. In 2014 her family moved her into a care facility.
Roz and the rest of the family remain dedicated to finding every opportunity to bring Angela joy. A few years ago, as Angela’s mobility was becoming limited, her family had the idea of giving her one last motorcycle ride. When she was young, riding with her dad on his motorcycle had been one of her favorite activities. Roz went on Facebook and asked if anyone had a sidecar they could borrow. Offers started pouring in. “The next thing we knew, 40 or 50 bikers got together and did a ride for Ang,” Roz says. Angela rode in the sidecar at the front of the procession. On the return journey, she rode in the back so that she could see all the people who had come out to support her, tears streaming down her face.
Today, Angela doesn’t speak much, though if she can get a single word out, her caregivers or family often know exactly what she needs. Roz brought a chalkboard to keep in her room, where Angela can point to a letter for the word she is trying to say; “yes” and “no” are written on opposite sides in nail polish. She relies on a wheelchair to get around.
Still, “where there’s music, there’s Ang in her chair and her legs are going,” Roz says.
“For me to stay positive, I’m always thinking about Ang. I have people ask me, How do you do it? And – you just do. Because you love, because you care, and you get through it.”
Patient resources
Huntington’s Disease Society of America is an organization founded with a mission to improve the lives of patients and families affected by Huntington’s disease.
