Shares of Pepgen Inc. (NASDAQ:PEPG) were trading at $3.36, up $1.99, or 145%, on word of initial positive data in myotonic dystrophy type 1 (DM1) from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I trial with PGN-EDODM1. The compound deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. The results exceeded Pepgen’s expectations for splicing correction after a single dose, officials said.

US lawmakers tell Kennedy: No more terminations

Citing recent executive orders that suggest additional cuts to the federal workforce may be in the offing, U.S. Sens. Patty Murray, D-Wash., and Jeanne Shaheen, D-N.H., wrote to Health and Human Services (HHS) Secretary Robert Kennedy urging him to end “indiscriminate cuts that will cause lasting harm to FDA’s public health mission,” to protect the agency’s statutory obligations, and to “prevent the stifling of American innovation.” According to the Friday letter, hundreds of probationary employees at the FDA’s device center and generic drugs office, as well as its tobacco center, were among the thousands terminated at the HHS early last week. Given that many of the FDA workers who were let go were in positions funded through user fees, the lawmakers said the firings raised “the alarm of whether [the Department of Government Efficiency], responsible for identifying probationary employees to fire, truly understands the nuances of funding at the FDA.”

Infectious disease index ends 2024 down 6.28%

The BioWorld Infectious Disease Index (BIDI) faced challenges throughout the year but narrowed its losses, ending 2024 down 6.28% after hitting a low of -25.12% in late April. In January, the BIDI rebounded slightly more, gaining 1.85%. Of note, shares of Novavax Inc. ended 2024 up 68%, Assembly Biosciences Inc. was up 60% and Cidara Therapeutics Inc. was up 28%.

Cosette paying $430M for Mayne's women’s health, dermatology franchise

Cosette Pharmaceuticals Inc. announced plans to acquire Adelaide, Australia-based Mayne Pharma Pty Ltd. for AU$672 million (US$430 million) to grow its women’s health and dermatology franchise in the U.S. Under terms, Cosette, of Bridgewater, N.J., will acquire 100% of Mayne’s shares at AU$7.40 per share in cash. The price represents a 37% premium to Mayne’s closing share price on Feb. 20. The deal is expected to close in the second quarter of 2025. “Today marks a pivotal moment in Mayne’s journey to improve patient access to life-enhancing medications,” said Mayne Pharma CEO Shawn Patrick O’Brien.

Eliminating redundancies opens up possibilities for protein engineering

Researchers have altered the genetic code in a strain of Escherichia coli, reducing the number of stop codons from three to one and assigning the freed-up stop codons to nonstandard amino acids. They reported on the recoded bacterium, which they named OCHRE, in Nature on Feb. 5, 2025. The engineered bacterium is “the first organism that’s been recoded where we’ve compressed redundant codons down to a single codon,” Farren Isaacs told BioWorld. “It’s exciting from a basic science perspective, a bioengineering perspective and an application perspective.”

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Acelyrin, Alumis, Alvotech, Atai, Atea, Blue Water Acquisition, BTB, Celltrion, Climb, Concentra, Diamedica, Elixirgen, Ensho, Firefly Neuroscience, Immunoprecise, Inhibikase, Johnson & Johnson, Meiragtx, Mesoblast, Nuclera, Psilera, Sensorion, Teva, Tivic, Werewolf, Xeris