Coming on the heels of an advisory committee in which the U.S. FDA and its independent advisers grappled with trying to fit an ultra-rare disease development program into the confines of the agency’s “significant evidence” requirements, an Oct. 16 public meeting on a Rare Disease Innovation Hub the agency is setting up seemed like a welcome step in the right direction for rare disease patients, their caregivers and companies working in the space.

While RNA-medicine developer Wave Life Sciences Ltd. brought in a clinical data win, it also got knocked back a step as a major collaborator will go its separate way. That didn’t stop Wave’s stock from standing strong on the day. The company’s ongoing phase Ib/IIa study of its A-to-I RNA editing oligonucleotide produced positive proof-of-mechanism data in treating alpha-1 antitrypsin deficiency, a rare, genetic condition that can lead to lung and liver disease.

When Andrew Wilks invented the JAK inhibitor momelotinib in the late 1980s for myelofibrosis, he never would have imagined it would take more than 20 years to develop and eventually be acquired for $1.9 billion. Today he’s on a mission to ensure Australian inventors have more options than he did, telling BioWorld that he had to sell the molecule for around $10 million because he couldn’t get funding.

Biopharma companies secured $16.31 billion in financing during the third quarter (Q3) of 2024, marking a 29% decrease from the $23.07 billion raised in Q2 and a 65% drop from Q1’s $47.25 billion. Despite the quarterly decline, year-to-date financings through September reached $86.63 billion, surpassing the total annual figures of both 2023 ($70.97 billion) and 2022 ($60.81 billion). Only the record-setting years of 2020 and 2021 saw higher amounts raised.