And They’re Off….. Watching the CAR-T Race

On March 29, Novartis announced that the US Food and Drug Administration had accepted the company’s BLA filings for CTL019 (tisagenlecleucel-T) for the treatment of pediatric and young adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia (ALL). Two… Read »

A Look at Duchenne Muscular Dystrophy: Part 3 – The Increasing Activism and Influence of Patient Advocacy Groups

On September 19, 2016 the US Food and Drug Administration approved Sarepta’s Exondys 51 (eteplirsen) injection, the first drug approved to treat the underlying cause of Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a… Read »

A Look at Duchenne Muscular Dystrophy: Part 2 – About DMD, and Approaches to Treatment, Old and New

Duchenne Muscular Dystrophy (DMD) is a rare genetic disease characterized by progressive muscle degeneration and weakness. It is one of the most frequent genetic disorders, affecting approximately 1 of 3,600 (primarily) male children. The disease is caused by a mutation… Read »

A Look at Duchenne Muscular Dystrophy: Part 1 – Marathon Pharmaceuticals, PTC Therapeutics, and Drug Pricing

In February of this year, Marathon Pharmaceuticals received approval for Emflaza (deflazacort) tablets and oral suspension for the treatment of patients over the age of five years with Duchenne muscular dystrophy (DMD), regardless of the particular genetic mutation associated… Read »

Win One/ Lose One: Latest Rulings in the IP Battles Over CRISPR/Cas9

On March 28, the latest round in the intellectual property battle over CRISPR/Cas9 ended with the European Patent Office (EPO) ruling in favor of the University of California / University of Vienna, the work of scientists Jennifer Doudna and Emmanuelle… Read »

Reflecting on AACR

The American Association for Cancer Research annual meeting is always a source of major news and discussion on the oncology research front, and the 2017 meeting held earlier this month was no exception.   One area of particular interest was… Read »

PARP Inhibitors Chalk Up Impressive Gains in Ovarian Cancer Maintenance Therapy

On March 27, the FDA gave Tesaro a broad approval for its PARP inhibitor, niraparib, as maintenance therapy for patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer after a complete or partial response to platinum-based chemotherapy. The… Read »

National Academies of Science and Medicine Cautiously Endorse Some Germline Gene Editing — In the Future

In February the National Academy of Sciences and the National Academy of Medicine issued a new report on the technical, social and regulatory concerns surrounding gene editing, entitled, “Human Genome Editing: Science, Ethics, and Governance.” Authored by a 22… Read »

Alzheimer’s Disease Part Three: Meeting 2025 Goals

In early 2011, the National Alzheimer’s Project was formed — a strategic plan aimed at addressing the looming health care crisis posed by the disease and the aging population. Its aim: to coordinate research efforts across the U.S. federal… Read »

Alzheimer’s Disease Part Two: Effecting Change – The Need for Validated Biomarkers of Risk and Response

We recently wrote about the continued, costly failure of Alzheimer’s disease drugs, often occurring in Phase 3 clinical development. Of 244 compounds tested from 2002 to 2012, only one gained approval. Today there are four compounds on the market for… Read »

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