Rare diseases
The 2025 meeting of the American Society of Hematology features some of the newest developments in blood cancers and rare diseases.
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.
The Phase III, open-label extension study suggests EPX-100 has a “positive” risk/benefit profile for the treatment of Dravet syndrome, analysts at H.C. Wainwright & Co. said Tuesday.
If approved, Ascendis’ TransCon CNP would become the second therapy for achondroplasia, challenging BioMarin’s Voxzogo.
This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question whether they will be enough to net their developers a significant share of the entrenched market.
Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator.
The regulator has received reports that a group of patients treated with Adzynma had neutralizing antibodies against the protein the therapy replaces.
Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company’s stock is down 80% on all the news.
The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed.
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