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In Brief This Week: Pramana, Vanderbilt University, MeiraGTx, Tikun, Atara

NEW YORK – Pramana, a company that develops AI-driven health technology, this week said its autonomous whole-slide imaging system will be used in Vanderbilt University Medical Center's pathology lab. Vanderbilt expects the system to advance precision medicine and improve clinical care and patient outcomes. The university also hopes it will enable new insights into disease, resulting in the development of innovative diagnostics and therapies. 


New York-based MeiraGTx this week said it has received rare pediatric disease designation from the US Food and Drug Administration for AAV8-RK-RetGC, a gene therapy it's developing as a treatment for Leber congenital amaurosis (LCA) caused by mutations in the GUCY2D gene. This marks the fourth rare pediatric disease designation that the company has received in recent months, including for other gene therapies in its ophthalmology pipeline for diseases such as LCA4 retinal dystrophy, Bardet-Biedl syndrome due to BBS10 mutations, and RDH12-associated retinal dystrophy. The rare pediatric disease designation is aimed at incentivizing development of new drugs for rare pediatric conditions by offering sponsors a priority review voucher they can use to expedite the review of other products.


The US Food and Drug Administration has granted Tikun Therapeutics orphan drug designations and rare pediatric disease designations for two drugs, namely rAAV2-U1a-hELP1, a gene therapy it's developing for optic neuropathy in familial dysautonomia, and BPN-36964, a small molecule splicing modulator it's developing for systemic treatment of familial dysautonomia, the New York-based company said this week. Tikun is a public benefit corporation that's wholly owned by the Familial Dysautonomia Foundation. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval. The rare pediatric disease designation is aimed at incentivizing development of new drugs for rare pediatric conditions by offering sponsors a priority review voucher they can use to expedite the review of other products.


The US Food and Drug Administration this week placed a clinical hold on Atara Biotherapeutics' investigational new drug (IND) application for T-cell immunotherapy Ebvallo (tabelecleucel) as a monotherapy treatment for adult and pediatric patients 2 years of age and older with Epstein-Barr virus (EBV)-positive post-transplant lymphoproliferative disease (PTLD). The FDA placed the clinical hold because of good manufacturing practices (GMP) compliance issues identified during the pre-license inspection of the third-party manufacturing facility. Earlier this month, the FDA also issued a complete response letter to Atara saying it could not approve Ebvallo in this indication after finding the issues in the third-party manufacturing facility. Atara said it has agreed with the FDA on the actions needed to release the clinical hold on Ebvallo. 


In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.