Second Anniversary for n-Lorem Foundation Marks Extraordinary Progress Toward Treating Nano-Rare Patients

n-Lorem’s donor and partner support has lowered per-patient cost by 40 percent enabling n-Lorem to expand its non-profit approach to personalized, investigational medicines for even more nano-rare patients.

SAN DIEGO, Calif.--(BUSINESS WIRE)--#ASO--n-Lorem, a nonprofit foundation, celebrates two years of progress in pioneering a novel non-profit approach to providing personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

“At n-Lorem, we are taking on the monumental challenge of targeting an individual’s unique genetic mutation by designing and developing a personalized experimental medicine just for them. We then provide that medicine for free, for life,” said Stanly T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “When I founded n-Lorem, I anticipated that we would be able to help a handful of patients. However, the demand has exceeded our expectations by ten- to twenty-fold. We now have more than 50 individualized drug programs, which represent more than 50 nano-rare patients, in development and plan to dose our first n-Lorem patient this summer. We anticipate adding more nano-rare patients this year, but as a non-profit, our growth and success are entirely dependent upon the contributions of our partners, donors and supporters.”

“We have this community of people who are just so supportive and who are fighting for a treatment for our daughter, Susannah. This gives us hope that a treatment will become a reality and that this personalized treatment might be able to change the course of Susannah’s life,” said Luke Rosen, father of Susannah and Founder of The KIF1A Foundation.

“We have made remarkable progress in growing our organization to meet the overwhelming demand from nano-rare patients and their families. Our team is driven to provide hope and help today and to explore ways to find more resources to help even more patients tomorrow,” continued Dr. Crooke. “We are very fortunate that patients, families, donors and partners are putting their trust in us. On behalf of the patients we serve, I thank each of you for your interest and support.”

Since its inception n-Lorem has:

• received and reviewed over 130 applications and accepted over 50 patients;
• advanced several programs toward treatment this year;
• established a network of 18 partners that provide support in all areas of drug discovery, development and manufacturing;
• received U.S. Food and Drug Administration (FDA) guidances that provide a supportive regulatory environment for ASO treatment of nano-rare patients;
• reduced per-patient costs of drug discovery, development and manufacturing; and
• built out its leadership team to support this rapid growth.

“We and our patients are grateful that in just two years our list of donors has grown to more than 75 and we have raised more than $50 million in funds, equipment and in-kind donations,” said Sarah Glass, Ph.D., Chief Development Officer of n-Lorem Foundation. “To meet this extraordinary demand, however, we must raise a great deal more in funds in the coming years. With the support of so many, we are confident that we will be successful.”

As part of its mission to improve the lives of patients, n-Lorem is implementing a ‘Patient Empowerment Program’. This podcast program will consist of interviews and lectures that will provide listeners the tools to understand complex drug discovery and development and create a community for nano-rare patients and their families to connect and share their stories.

Learn more about the exciting progress n-Lorem has made by reading Stan Crooke’s annual letter

• Read: Annual Letter

Learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
• Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 130 applications for treatment with more than 50 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn, YouTube and Instagram.

n-Lorem Contact:
Amy Williford, Ph.D.
Director of Communications
amy.williford@nlorem.org