Biopharmaceutical executives, especially those who have founded their own companies, never cease to amaze me. Why? Well for starters, the process of starting with nothing but an idea and then pulling together all the necessary resources to build a company capable of developing and delivering a product to patients is an amazing logistical challenge. It requires someone capable of big-picture thinking. Someone confident in being able to recruit top talent to join them on their journey.

In the early 2000s, chimeric antigen receptor T (CAR-T) cells emerged on the scene with promise as a revolutionary cancer treatment. However, several key challenges and, in particular, downsides related to manufacturing, continue to hamper broad adoption of this therapeutic approach. In addition, existing CAR-T cell therapies are based on autologous approaches (i.e. using the own patient’s cells), meaning that product has to be individually made for each patient. This presents a true challenge in terms of scalability and having the product available for a large patient population.

Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics. Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene. With the designation, the drug will receive priority review by the FDA, and it could qualify ProQR for a voucher redeemable for priority FDA review of a subsequent marketing application for a different product, the release said.

At a medical mycology meeting here earlier this month, scientists presented promising phase II clinical data for rezafungin, a new member of an existing class called the echinocandins developed by Cidara Therapeutics in San Diego.

Arrowhead Pharmaceuticals stock flirted with a breakout Friday after the biotech company said it’s experimenting with drugs that will “silence” multiple troublesome genes simultaneously. Previously, that was impossible, Chief Executive Christopher Anzalone said late Thursday on CNBC’s Mad Money. Arrowhead Pharmaceuticals (ARWR) is among a slew of biotech companies using technology called RNA interference to silence genes responsible for creating problematic proteins.