21
Sep
2020
Tag: rare diseases
13
Apr
2020
Bionest
FDA Finalizes Gene Therapy Guidelines and Adds New Draft Guidance on Orphan Drug Exclusivity
The field of gene and cell therapy is flourishing. How is the FDA responding?
23
Dec
2019
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.
10
Jun
2019
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...
25
Feb
2019
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...
18
Dec
2018
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...
15
Oct
2018
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...
8
Oct
2018
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...
17
Sep
2018
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 —...
17
Jul
2017