With more than 70,000 people living with cystic fibrosis (CF) worldwide, according to the Cystic Fibrosis Foundation, the introduction of CF transmembrane conductance regulator (CFTR) modulator therapies has revolutionized treatment of the disease. However, these drugs are not effective for around 10% of CF patients, driving a significant unmet therapeutic need. One startup hoping to address this is Anoat Therapeutics.
Emerging from stealth mode, new immuno-oncology company Dotbio Pte. Ltd. closed an oversubscribed $5.6 million pre-series A round to accelerate development of its multifunctional and intracellular antibody therapies.
Alltrna Inc. raised $109 million in a series B round to back its work with transfer RNA, with the first targeted indication of stop codon disease: a condition that takes in thousands of rare and common afflictions stemming from nonsense mutations where the amino acid code has been mutated into a “stop” codon, also known as a premature termination codon.
South Korean biopharma Connext Co. Ltd. is set on outdoing standard-of-care therapies for Dupuytren’s contracture. The Daegu-headquartered Connext recently secured U.S. FDA IND approval for a phase I/II trial on its recombinant collagenase clostridium histolyticum, called CNT-201, inching closer to its goal of providing an affordable but high-quality therapeutic option for patients with the rare, progressive connective tissue disorder.
When looking for a name, the founders of startup Nexo Therapeutics Inc., which recently emerged from stealth mode with a $60 million series A financing and a research collaboration with the University of Texas MD Anderson Cancer Center, sought a moniker that truly captured its approach to drug development.
New company Amber Bio emerged on Aug. 3 with $26 million in seed funding that will help advance an RNA-based gene editing platform that leverages Cas-based systems to create safer medicines. Through the company’s platform, a single drug can be used to treat diseases with high allelic diversity. The company plans to develop its own genetic medicines internally, while also licensing out the technology to expand its reach.
Precision medicine startup Solu Therapeutics has raised $31 million in an oversubscribed seed round to advance a therapeutic candidate based on technology that identifies cell surface, tumor-associated targets that antibodies alone fail to latch onto. The company was founded by venture capital firm Longwood Fund and has high hopes for its cytotoxicity targeting chimera platform. “[It] has the potential to unlock new tumor-associated antigens and develop molecules that deplete pathogenic immune cells and extend the half-life of small-molecule antagonists and agonists,” CEO and co-founder of Solu, David Donabedian, told BioWorld.
Viage Therapeutics Inc. – formerly Digestome Therapeutics Inc. – on July 26 unveiled positive data from its phase I study on DGX-001 for mild cognitive impairment in Alzheimer’s disease (AD) and Parkinson’s (PD). The randomized, double-blind, placebo-controlled study demonstrated changes in brain activity according to quantitative electroencephalography (qEEG) measurements in 68 healthy volunteers dosed with single ascending and multiple ascending doses.
Newco Renaissance Pharma Ltd. has been formed to take a new treatment for neuroblastoma developed at St Jude’s Children’s Research Hospital through to market. The product, Hu14.18, has been in-licensed following a phase II trial conducted at the hospital in which patients with newly diagnosed high-risk neuroblastoma had a three-year event-free survival of 73.7% and an overall survival of 86%.
The radiopharmaceuticals field is gaining traction as companies realize that by harnessing the power of radioactive atoms, they can create targeted and more effective cancer drugs which cause less damage to healthy tissues. As a result, competition for a place as a leading radiopharma firm is high, and the market is estimated to reach $11.5 billion by 2027.