21
Nov
2022
Tag: rare diseases
13
Dec
2021
Bionest
UK Policy Changes Allow Earlier Access to Innovative Medicines for Rare Diseases
Access to new treatments for rare diseases in the UK has often been a challenge. Will a new decision-making framework & funding improve the situation?
21
Sep
2020
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.
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FDA Finalizes Gene Therapy Guidelines and Adds New Draft Guidance on Orphan Drug Exclusivity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