Advances and Set Backs for CAR-T Therapy

Late August and early September was a period of big news — both positive and negative — on the CAR-T front. On the positive side, Novartis’ received the first FDA approval of a CAR-T therapy for Kymriah (tisagenlecleucel), for the… Read »

Diagnosing Alzheimer’s Disease — Part 2

As we wrote in our last post, the ability to accurately diagnose Alzheimer’s disease before a person’s death has been problematic. Experts estimate that a third or more of patients’ cognitive impairment or dementia may be inaccurately diagnosed. As a… Read »

New Developments on the Alzheimer’s Disease Diagnostic Front — Part I

In July, we attended the Alzheimer’s Association International Conference in London. The big news at the meeting was on the diagnostics front.  In the aftermath of the recent string of trial failures, diagnostic tools have become a critical area of… Read »

Recent Developments in IO – Our Take on a Couple of Key Events

On July 27, AstraZeneca (AZ) disappointed their investors and many immuno-oncology watchers with the results of their MYSTIC trial combining two immunotherapies targeting PD-L1 (durvalumab/Imfinzi) and CTLA-4 (tremelimumab) in newly diagnosed Stage-IV lung cancer patients.  The AZ combination therapy failed… Read »

Pointed Brexit Warnings from the Pharmaceutical and Biotech Industry

It has now been a year since the United Kingdom voted to leave the European Union. Since then, discussions about the process of achieving Brexit have been fierce — including those affecting the pharmaceutical and biotechnology industries where business is… Read »

Human Germline Gene Editing Becomes Reality — at Least at the Research Stage

Back in April of this year, we wrote about the National Academies of Science and Medicine cautiously endorsing the possible use of gene editing at the germline level to correct serious diseases or medical conditions. Their report commented that scientists… Read »

CAR-T Nears the Marketplace, with Unanimous Approval Recommendation – but Challenges Remain

On July 12, Novartis’ CAR-T therapy, CTL019 (tisagenlecleucel), received a unanimous recommendation for approval from the reviewing U.S. Food and Drug Administration’s advisory panel for use in the treatment of children and young adults with advanced acute lymphoblastic leukemia (ALL)…. Read »

Gene Therapy: Coming of Age Perhaps, but Who Will Pay?

New technologies tend to go through cycles of interest and attention. Initial excitement about the potential of a technological advance too often builds to over-blown hype, followed by a quick reversal of interest in the aftermath of a big disappointment…. Read »

CRISPR Reaches the Clinic

Sometime mid-month, the first clinical trial employing CRISPR gene editing technology within the human body will initiate in China, at Sun-Yat Sen University. Scientists there will seek to use CRISPR and TALEN, another gene editing method, to treat persistent HPV… Read »

FDA: Thinking “Outside the Box”

On May 17, the U.S. Food and Drug Administration granted an expanded approval to Vertex’s Kalydeco (ivacaflor), tripling the number of mutations in the cystic fibrosis (CF)-associated gene CFTR that the drug can treat, from 10 to 33. The agency’s… Read »

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