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. Read More
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. Read More
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. Read More
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. Read More
A new study helps explain the role of genetic variation in shaping gene regulation in the Indonesian archipelago, one of the most diverse regions in the world. “This study is the only study of splicing from Southeast Asian populations. There is basically no data from this part of the world,” study author Irene Gallego Romero told BioWorld. For drug discovery, most of the people that have historically participated in clinical trials are of European ancestry, and scientists are just beginning to study African populations to better understand genetic differences in these populations, said Romero, a population geneticist and biological anthropologist at the University of Melbourne. Read More
Shares of Novavax Inc. (NASDAQ:NVAX) dropped nearly 20% Oct. 16 to close at $10.15 after the company reported a serious adverse event had prompted a U.S. FDA clinical hold for its COVID-19-influenza combination and standalone flu vaccine candidates. Read More
Backed by AI technology, Aigen Sciences Inc. raised ₩12 billion (US$8.8 million) in a series A financing round to further advance its cancer and rare disease drug pipelines. Read More
New hires and promotions in the biopharma industry, including: Acelyrin, Achieve, Adimab, Adverum, C4, Candid, Keros, Myrobalan, Spinogenix, Volastra. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Amgen, Argenx, Cartesian, Celltrion, Eupraxia, Janssen, Meiragtx, Thryv, Topas, Ventyx. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alleo Labs, Atavistik, I-seq, Glaukos, Isomab, Molecure, Notable Labs, Ocean, Pfizer, Ripple, Ubiquigent, Vic. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Almirall, Immuneering, Intrabio, Junshi, Lantern, Neurosense, Novartis, PTC Therapeutics, Sellas, Topalliance. Read More
