We’re addressing perhaps the most difficult decision n-Lorem has to make in our work, declining a patient application. Today, our acceptance rate is ~59%. These decisions are made with heavy hearts at n-Lorem, and we are aware of how heartbreaking the decision to decline an application may be for a patient and their family. In this episode, we’ll discuss the main reasons a patient’s application may be denied, including safety, geography, and other factors. 

• 1:43 – Non-technical issues: Outside of USA, inability to find treating physician, patient population size, if the patient is being treated by another experimental medicine, including repurposed drugs

• 7:15 – Definitions to better understand the conversation: Heterozygous mutation, haploinsufficiency

• 9:40 – n-Lorem must understand the cause of the disease, the nature of the mutation, and n-Lorem must be confident that the ASO designed would deliver value to the patient

• 12:35 – n-Lorem must be confident that only a single gene is causing phenotypes, and that the target is not a null mutation

• 13:37 – n-Lorem may accept patients, but then discontinue a program if the patient cells are unable to be grown for lab use and testing

• 14:56 – For patients requiring an allele-selective ASO, n-Lorem must identify non-pathogenic mutations to attempt to create an ASO capable of helping the patient

We cannot do
this alone

We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.