The FDA has granted orphan drug designation to Modalis Therapeutics Corp.’s MDL-101, a novel epigenetic editing therapy being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), a severe, early-onset muscular dystrophy caused by the absence of the LAMA2 protein.

The abscopal effect occurs when therapy leads to the regression of metastases distant from the target tumor. In the current study, researchers from INSERM aimed to evaluate the abscopal effect potentially inducted by the use the targeted radionuclide therapy (TRT) candidate, [131I]ICF-01012, which has been previously shown to target only pigmented metastases.

Currently, cancer therapy trial-and-error methodology is inefficient and unsustainable. Oncology is the worst therapeutic area for drug trial success; only 3.4% of drugs that enter phase I end up being FDA approved, and 57% fail due to poor drug efficacy in trials. Building tools that may aid in predicting an individual’s response to a specific therapy may help in reducing costs, guesswork, and importantly improve the outcome of patients and accelerate new drug development.

Modifi Biosciences Inc. has come a long way very quickly. Spun out of Yale University in 2021, it published data on DNA in Science in July 2022, and now it’s being acquired by Merck & Co. Inc. for $30 million up front. Ultimately, Modifi shareholders could receive milestones of up to $1.3 billion.