Expected cash runway extended into Q3 2027, beyond multiple potential inflection points including Dyne’s first planned commercial launch in early 2027 - ...

Expected cash runway extended into Q3 2027, beyond multiple potential inflection points including Dyne's first planned ...

Avidity Biosciences (RNA) experienced a remarkable share price surge of ~900% from late 2023 to mid-2024. This is mainly due to the progress of three oligonucleotide candidates indicated for DMD ...

Many FDA accelerated approvals continue to rely heavily on surrogate endpoints, raising ongoing uncertainty about the true clinical benefits of these therapies.

The CRL was given because the FDA did not consider the phase 1/2 IGNYTE trial to be an adequate and well-controlled clinical ...

Targeted Oncology connects oncology professionals with updates on immunotherapy, biomarkers, cancer pathways, and targeted precision medicine strategies.

The US Securities and Exchange Commission (SEC) has approved the conversion of Bitwise 10 Crypto Index into an ...

FDA confirmed that eGFR slope is an acceptable surrogate endpoint for accelerated approval of rilparencel in patients with CKD and type 2 diabetesFDA also confirmed ...

Among reasons for the rejection, the agency cited a lack of US patient representation in the STARGLO trial assessing ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

The goal with ProKidney's rilparencel-branded therapy is “to demonstrate the therapy’s potential to preserve kidney function ...

The FDA recently unveiled a new national priority voucher program that would seek to trade faster drug approvals for lower ...