With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...

The Avestagenome Project® International Pvt. Ltd. (AGENOME) & Avesthagen Limited, in partnership with the Tata Institute for ...

Percheron Therapeutics Ltd has submitted a non-binding proposal to an international pharmaceutical company to in-license a new drug development program focused on a rare neurological disease.

Inozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. We are ...

Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a ...

We recently published a list of the 10 Oversold Pharma Stocks to Buy According to Analysts. In this article, we are going to ...

Strong evidence for ALS often comes from familial forms of the disease, where specific gene mutations— for example in the ...

We recently published a list of the 10 Best Performing Pharma Stocks So Far in 2025. In this article, we are going to take a ...

We recently published a list of the 10 Best Performing Pharma Stocks So Far in 2025. In this article, we are going to take a ...

The U.S. Food and Drug Administration (FDA) has announced its recent approval of Ctexli (chenodiol), marking the first-ever treatment for cerebrotendinous xanthomatosis (CTX), a rare and debilitating ...

Every Cure was co-founded by a Penn immunologist after discovering an off-label treatment for his own rare disease.

Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and ...