Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.

The year 2025 proved especially active with regard to deals that centered on the indication previously known as nonalcoholic steatohepatitis (NASH).

In a threshold event in the U.S., Medicare is planning to break through its obesity coverage barrier with a voluntary test of a model designed to enable Medicare Part D plans and state Medicaid programs to cover GLP-1 drugs prescribed for weight management.

Needle-phobic obesity patients got their first workaround with the U.S. FDA clearance of Novo Nordisk A/S’ once-daily GLP-1 Wegovy (semaglutide) pill, the first of its kind.

Enveda has obtained IND clearance from the FDA and commenced dosing in a phase I study of ENV-308, a once-daily oral therapy designed for chronic weight management.

Biomarin Pharmaceutical Inc. followed up its May takeover of Inozyme Pharma Inc. with a much larger agreement to acquire Amicus Therapeutics Inc. for $14.50 per share in an all-cash transaction, tipping the equity-value scale about $4.8 billion.

Fat tissue balances energy by storing lipids during times of abundance and mobilizing them when needed, yet sustained metabolic stress demands mechanisms that limit excessive lipid loss. Researchers at the University of California San Diego (UCSD) report that stress-induced fat breakdown triggers a neutrophil response in visceral adipose tissue that feeds back to restrain lipolysis and preserve lipid reserves.

Eli Lilly and Co. took another step toward adding a way for patients to hang onto their weight loss when the firm disclosed positive top-line data from the phase III Attain-Maintain trial with orforglipron, a once-daily oral small molecule GLP-1 receptor agonist. Indianapolis-based Lilly said that at one year orforglipron met the primary and all key secondary endpoints vs. placebo, delivering superior weight maintenance as an adjunct to a healthy diet and physical activity, using the efficacy estimand and modified treatment-regimen estimand.