17
Apr
2023
Tag: gene therapy
21
Nov
2022
Bionest
Competitors Collaborate to Understand Shared DMD Adverse Event
Adverse responses to experimental gene therapies for Duchenne muscular dystrophy leads to unique collaboration between pharma competitors.
10
Oct
2022
Bionest
Are the Blues Ending for Bluebird?
Gene therapy developer bluebird bio may be turning a corner, with recent FDA approvals for two of its rare disease therapeutics.
27
Dec
2021
Bionest
Addressing Gene Therapy Uncertainties
Are we close to more streamlined and standardized approaches to gene therapy testing and regulations?
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FDA Finalizes Gene Therapy Guidelines and Adds New Draft Guidance on Orphan Drug Exclusivity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?
2
Mar
2020
Bionest
New Treatment Options for Hemoglobin-Based Blood Disorders
Sickle cell disease is a group of inherited disorders that affects hemoglobin, the oxygen-carrying protein in red blood cells (RBCs), and causes these cells to distort in shape at low oxygen levels. This condition affects 1 in 500 Americans of African descent and 1 in 1,000-1,400 of Hispanic descent, as well as millions...
2
Sep
2019
Bionest
Zolgensma Launch “On Track,” but Significant Challenges Remain
On July 18, Novartis told analysts its Zolgensma launch was “on track.” Despite the gene therapy’s $2.1 million price tag, the company states the launch is “one of the most successful launches from an access standpoint in rare diseases.” Novartis said it had reached coverage deals with more than 20 commercial insurance plans, covering approximately 40%...
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...
18
Feb
2019
Bionest
One-and-Done Pricing Challenges
As more gene and cell therapies come to market, a big challenge facing both drug developers and payers is designing and instituting appropriate pricing models for these one-time, potentially curative treatments. Innovation should be rewarded, and a curative — or at least, very long-lasting — treatment is likely to save significant dollars in the typical...
15
Oct
2018