Bionest

Look Beyond the US and Europe for Rare Disease Launches

Rest-of-world markets offer good opportunities for rare disease companies to expand their sales potential beyond what U.S. & E.U. markets offer, and the requirements for market entry should be considered at a high level early on.

Bionest

The SMA Market: Assessing the Unknowns

Challenges and opportunities in the field of spinal muscular atrophy, including how new disease-modifying therapies are likely to change SMA’s course over time.

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

Artificial Intelligence and Image-Based Diagnostic Tools

We have recently been hearing much about “machine learning” and “artificial intelligence” (A.I.). While many tend to use the two terms almost interchangeably, there is a distinct difference between the two.   Machine learning, also known as “deep learning,” is a field of applied mathematics that uses algorithms and statistical models to build predictive systems...

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

Gene Therapy Update Part 2

Last week we wrote about recent news in the field of CRISPR/Cas9 research. But CRISPR is not the only approach to gene editing under development, nor the most advanced.   First Human Gene Editing Data — But Not from CRISPR   Sangamo Therapeutics, Inc. has been developing an approach to gene editing based on zinc...

Bionest

Gene Therapy Update Part 1: Big Advances and Big Questions for CRISPR

The gene therapy field has seen several developments in recent months, especially around CRISPR, with both significant achievements and further controversies about the safety of the gene editing approach.   CRISPR Cure for Duchenne’s Muscular Dystrophy (DMD) in Dogs   Eric Olson’s team of researchers at the University of Texas Southwestern’s Harmon Center for Regenerative...

Bionest

FDA Issues Set of Guidelines on Gene Therapy

Back in December, the U.S. Food and Drug Administration (FDA) granted its first approval for a gene therapy product, Spark Therapeutics’ Luxturna. According to the Alliance for Regenerative Medicine, as many as 50 more such treatments — addressing a diverse range of health problems from genetic, autoimmune, and heart diseases, to cancer and HIV/AIDS —...

Bionest

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. But if continued research later produces truly encouraging results, interest can build again as time...