When CEO Avanish Vellanki founded and incorporated Rain Therapeutics on his living room couch in the spring of 2017, there was no drug in development, no money, and a team of only four people. Five years later, after halting the development of its first licensed product, the Newark, CA-based precision oncology company awaits topline results from a pivotal Phase 3 trial in liposarcoma, which could open the door to a cancer therapy — milademetan — capable of treating as many as half of all known cancer types.

“Like the ongoing activities that financial institutions have adopted regarding [their] intentional efforts to be inclusive, the life sciences industry may want to adopt similar approaches to increase the awareness of career opportunities within the field. 

MeiraGTx today announced top-line data from the Phase 1/2 clinical study MGT009 (NCT03252847) of botaretigene sparoparvovec (formerly referred to as AAV-RPGR), an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP) with disease-causing variants in the RPGR gene.

The experimental Arrowhead Pharmaceuticals drug fazirsiran can reduce the accumulation of a mutant protein by 83% among people with alpha1-antitrypsin deficiency (AAT) disease, according to results from an open-label phase 2 trial involving 16 volunteers.

The biggest challenge that the pharma and biopharma industries currently face is unwanted immunogenicity.

We see huge potential for growth in precision medicine, particularly precision oncology, in the near future.

New data and a new documentary called “The Human Trial” together illuminate the hard work, sacrifice, and slow, iterative progress in the long search for a biological cure for type 1 diabetes.

Rather than target mutated p53, Rain Therapeutics is looking at normal p53 expression as part of its novel mouse double minute 2 homolog (MDM2) inhibitor therapy. In the coming 12 months, Rain expects to have an interim readout for a Phase II study of its liposarcoma therapy, called milademetan (Rain-32), and a pivotal readout for its Phase III trial of the same compound.