Bionest

Real World Evidence Replacing Some Clinical Trials?

Pharmaceutical companies have long used real-world data on drug performance to help justify their pricing decisions to payers. But now such real-world evidence (RWE), gathered from electronic medical records (EMRs), national patient registries and other patient databases, is increasingly being looked at as a way to make the case for new supplemental approvals and label...

Bionest

Highlights from AAN: Part 1

The Bionest team attended the American Academy of Neurology (AAN) in early May, where we heard exciting data presented in a number of neurological conditions. Among the highlights of this year’s conference:   Transformative Success in Spinal Muscular Atrophy   Spinal muscular atrophy (SMA) was a hot topic at AAN, with presentations of positive clinical...

Bionest

Breaking Out of the Bubble

X-linked severe combined immunodeficiency (X-SCID, or SCID-X1), is an orphan monogenic disorder popularly known as ‘bubble boy disease’ that affects 1 in 50,000-100,000 live births. The most serious of all primary immunodeficiencies, X-SCID causes severe failure of the immune system, including a lack of T cells, natural killer (NK) cells, or functional B cells. For...

Bionest

Meet You at ASCO!

ASCO, the world’s largest meeting focused on cancer, is always one of the highlights of our calendar here at Bionest. This year is no exception and several of us are headed to Chicago for this year’s event, which is being held from May 31 to June 4. If you are going to ASCO, we’d love...

Bionest

Nobel Award Winning Science Reaches the Clinic — Finally

In 2006, Japanese scientist Shinya Yamanaka turned back the biological clock of mature mouse cells, reprogramming them into stem cells capable of becoming any cell types. This accomplishment led to his sharing a Nobel Prize in Physiology or Medicine in 2012 with Sir John Gurdon, another pioneer in the field of regenerative medicine. The scientific...

Bionest

Targeting Brain Cancer with New Vaccine Approaches

Glioblastoma multiforme (GBM) is the most common form of brain cancer affecting adults. It is also one of the deadliest and hardest to treat cancers. About half of all people with GBM survive longer than 18 months, but only 15% are still alive five years post diagnosis. Of the approximately two dozen experimental treatments tested...

Bionest

Multiple Myeloma: The View from ASH 2018

Multiple myeloma (MM) is a deadly bone marrow cancer. In recent years, the treatment landscape for this disease has improved considerably. However, most patients still relapse and the need for new and better therapeutic options endures.   Encouraging news about several approaches were reported at the American Society of Hematology (ASH) in December. Results from...

Bionest

Neuromuscular Drug Development Summit

At the end of October, we attended the inaugural Neuromuscular Drug Development Summit. This new meeting brought together thought leaders from industry, academia, and the patient advocacy community to discuss scientific progress and issues affecting companies developing drugs for these mostly rare diseases.   Neuromuscular diseases — such as muscular dystrophies, motor neuron diseases, mitochondrial...

Bionest

Report on ESMO 2018 – Part Two

We recently returned from the European Society for Medical Oncology (ESMO) meeting where conference news highlighted the increased movement of targeted therapies and immunotherapies into adjuvant and early-stage cancer settings. Read our earlier report on combination therapy trials in melanoma, colon cancer and breast cancer, including triple-negative breast cancer. Here, we cover other targeted therapies....

Bionest

Report on ESMO 2018 – Part One

We recently returned from the European Society for Medical Oncology (ESMO) meeting where conference news highlighted the increased movement of targeted therapies and immunotherapies into adjuvant and early-stage cancer settings, plus plenty of positive data from a variety of combination therapy trials in breast cancer, including triple-negative breast cancer.   (Neo)Adjuvant Treatment Gets a Boost  ...