Team CHM’s World Champion

What length with a mother go to try and save her son’s sight? For Stephanie Sims that answer is 140.6 miles of swimming, biking and running at the 2017 IRONMAN World Championships in Kona, Hawaii.

Stephanie Sims competes in IRONMAN Wolrd Championship for Team CHMStephanie’s IRONMAN World Championship journey began in 2016, when she raced on Team CHM’s IRONMAN Wisconsin team, earning her entry to the coveted Kona race. For the last two years, Stephanie has spent countless hours training for these races and raising funds in support of the Choroideremia Research Foundation.

Stephanie’s journey to Kona on behalf of Team CHM was personal. She is a carrier of choroideremia and in addition to her father and other family members, Stephanie’s son Sullivan has CHM. Stephanie is racing for a reason: to make a difference in the fight to save Sullivan’s sight and the sight of others with CHM.

Stephanie, thank you for being our champion. Because of you, we are closer to finding treatment and a cure for CHM.



Nightstar Therapeutics raises $75 Million in IPO to fund Pivotal Phase 3 Gene Therapy Study

Nightstar Therapeutics held it’s Initial Public Offering on Thursday September 28th, raising $75 Million which will be used to advance the company’s gene therapy treatment for Choroideremia along with other Retinal Degenerative Diseases the company is working on.  The company, which was spun out of Oxford University has completed Phase 1/2 Clinical Trials and is preparing to undertake Pivotal Phase 3 trials for their gene therapy treatment targeting Choroideremia.  The Nightstar treatment involves the use of an adeno-associated viral (AAV) vector to deliver a corrected version of the Gene that causes Choroideremia.  Because gene therapy treatment is aims to replace the defective gene with a corrected version of the gene, the treatment is designed as a one time injection.

Choroideria Research Foundation President Dr. Christopher Moen and wife Alis Moen were invited to attend the NASDAQ Closing Bell Ceremony by Nightstar CEO David Fellows.  Dr. Moen and the Choroideremia Research Foundation were recognized on stage during the Closing Bell ceremony.  The Choroideremia Research Foundation provided grants in support of the early work performed by Dr. Miguel Seabra which laid the foundation for the development of the Nightstar Therapeutics gene therapy treatment, and has provided support for lead surgeon and Nightstar Co-Founder Dr. Robert MacLaren.

For more information on Nightstar Therapeutics and their gene therapy development you can visit their website by clicking here.  Nightstar Therapeutics is now being traded on the NASDAQ under the symbol NITE.