Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

XTalks

JANUARY 28, 2022

Uveal melanoma is a rare and aggressive type of melanoma of the eye. Up to 50 percent of patients will develop metastatic disease, mainly spreading to the liver, making treatment difficult and limited. The new treatment will thus help address the unmet need for the disease. The median treatment time is about 23 weeks (or 5.3

FDA Approval 98

FDA Approval Development Licensing Licensing 98

pharmaphorum

APRIL 5, 2022

Uveal melanoma is a rare cancer of the eye, which in the majority of patients is localised. mUM is the most common form of cancer affecting the eye in adults, but is still rare, diagnosed in a around 1,700 patients each year in the US, with a few hundred already with metastatic disease that has spread to other tissues.

Protein 52

Protein Sales Drugs Trials 52

pharmaphorum

OCTOBER 21, 2021

Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug. Biogen stumped up $1.525 billion upfront for rights to the drug and another Sage candidate for neurological diseases last year, when the expectation was that Aduhelm was unlikely to get FDA approval.

Sales 118

Sales Doctors Doctor Gene Therapy 118

pharmaphorum

JUNE 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8

Gene Therapy 59

Gene Therapy Gene Marketing Trials 59

pharmaphorum

AUGUST 18, 2022

.” The gene therapy specialist’s chief executive Andrew Obenshain said the approval “marks a watershed moment for the field of gene therapy” and is “ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

The Pharma Data

MARCH 16, 2022

using SNAP-tag) involves labeling cell proteins with a fluorescent dye, thereby enabling researchers to observe how proteins interact in the living cell and where in the cell they are located. “We His latest work on the development of biosensors has huge potential to benefit patients with certain metabolic diseases.”.

Protein 52

Protein Scientist Gene Development 52