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Life Sci Turns 5

THIS YEAR 

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A Network And Pivot Model For Success In Cancer Drug Development

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.

biospace

Beyond Increments: Black Perspectives on Representation in the Life Sciences

“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. 

opthalmology times

MEIRAGTX ANNOUNCES DATA FROM PHASE 1/2 CLINICAL STUDY OF GENE THERAPY FOR PATIENTS WITH X-LINKED RETINITIS PIGMENTOSA

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.

Drug reduces mutant protein that can lead to fibrosis in rare genetic liver disease

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.

pharmas almanac

What do you consider the biggest challenge that the pharma/biopharma industry currently faces?

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

pharmas almanac

IN WHICH ORPHAN SPACES OR UNMET MEDICAL NEEDS DO YOU THINK WE WILL SEE THE NEXT TREATMENT INNOVATIONS?

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

NEW DATA, FILM HIGHLIGHT ISLET CELL TRANSPLANTATION PROGRESS

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.

biospace

RAIN TARGETS NORMAL P53 EXPRESSION WITH POSSIBLE PAN-CANCER THERAPEUTIC

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.