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The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.

Takeda and Denali Therapeutics first partnered in early 2018 to advance drugs for neurodegenerative diseases. One asset, for Alzheimer’s disease, was previously discontinued after an FDA hold and disappointing early data.

After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination of 20 years of hard work unraveling the mysteries of the blood-brain barrier.

Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.

The deals keep rolling in, with Lilly penning a $7 billion pact for gene delivery biotech Kelonia Therapeutics and UCB taking over cell therapy-focused Neurona Therapeutics; President Trump signed a new executive order supporting the development of psychedelic therapies, sparking fanfare and concern alike; and the FDA’s recent Replimune decision has triggered broader debate about the agency’s flexibility.