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After two years characterized by layoffs, pipeline reorganizations, FDA delays and clinical holds, Novavax CEO John Jacobs says the company is at a pivot point.

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.

Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.

The Japanese pharma had one asset rejected by the FDA and withdrew a regulatory application for another, but already this month the company has secured an approval for AstraZeneca-partnered Dato-DXd, to be marketed as Datroway.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.

Less than a day into his second term, President Donald Trump ordered a freeze on communications at major public health agencies, among other moves that have sent waves through the biopharma industry.

At J.P. Morgan, most biopharma executives expressed a neutral stance on the incoming administration, but just days later, President Trump issued multiple executive orders that concern the industry.

Five years ago, Gilead signed a massive deal with Galapagos. After a restructuring, the pharma is still hunting for the potential it saw at the original signing.

There are currently no treatments available for celiac disease beyond a gluten-free diet. Several late-phase companies aim to change the paradigm and deliver hope and progress soon.

As the year gets underway, analysts and biotech executives highlight cell therapy’s pivot from oncology to autoimmune diseases, a continued appetite for next-generation obesity drugs and an increased focus on neuromuscular, kidney and cardiovascular diseases.