Vertex Pharmaceuticals Archives - Medical Design Sourcing

100 top cell and gene therapy companies to watch in 2023

[Image courtesy of ipopba/Adobe Stock]

The cell and gene therapy sector is poised to deliver a wave of new therapies that could potentially cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023.

While manufacturing and regulatory challenges remain, the cell and gene therapy industry continues to offer significant therapeutic potential and commercial opportunities.

This year, we have beefed up our list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.

In selecting the companies for this list, we weighed public companies’ degree of involvement in cell and gene therapy, including drug developers, equipment vendors and CDMOs specializing in the area. For private companies, we factored in their funding levels, pipeline and degree of history for the list.

B…

Gilead Sciences, Merck near the top of Newsweek’s most responsible companies list

A handful of big names in drug discovery and development are among the 500 “most responsible,” according to Newsweek.

The outlet published its “America’s Most Responsible Companies 2022” list, marking the third installment of the compilation (in partnership with Statista), this time expanded to include 500 of the largest public corporations around. Companies were judged with an overall score out of 100 that combined assessments on environmental, social and corporate governance performance.

Gilead Sciences (NSDQ:GILD), Merck (NYSE:MRK) and Pfizer (NYSE:PFE) were among the pharmaceutical companies to land a spot in the top-500, with a number of other drug development, pharmaceutical processing and manufacturing companies included.

The highest-ranking company was Gilead in sixth, landing an overall score of 89.9 points (90.6 environmental, 81.1 social and 98.2 corporate governance).

Merck was next-best in ninth at 89.3 points (94.6, 81.4, 92.…

Pharma’s top 20 R&D spenders in 2020

[Lab image by Michal Jarmoluk from Pixabay]The past year has been an unprecedented time for the pharmaceutical industry.

On the one hand, the pandemic resulted in substantial delays to clinical trials while also forcing sponsors to rethink clinical trial design to protect participants. But on the other hand, the pandemic underscored the importance of the pharma industry in society.

While the pandemic certainly was a driver for substantial R&D spending in the industry in 2020, a significant expense for many companies last year was licensing fees and other acquisition costs. That trend is not just apparent for Incyte, which tops this list, but also for several other companies in this ranking of 20 firms, which are ranked below based on the percent of revenue they invest in R&D. (To find out the top 50 pharma companies, check out our recently published ranking here.)

Get the full picture from our sister site, Drug Discovery & Development.

Pharma’s top 20 R&D spenders in 2020

[Lab image by Michal Jarmoluk from Pixabay]

The past year has been an unprecedented time for the pharmaceutical industry.

On the one hand, the pandemic resulted in substantial delays to clinical trials while also forcing sponsors to rethink clinical trial design to protect participants. But on the other hand, the pandemic underscored the importance of the pharma industry in society.

While the pandemic certainly was a driver for substantial R&D spending in the industry in 2020, a significant expense for many companies last year was licensing fees and other acquisition costs. That trend is not just apparent for Incyte, which tops this list, but also for several other companies in this ranking of 20 firms, which are ranked below based on the percent of revenue they invest in R&D. (To find out the top 50 pharma companies, check out our recently published ranking here.)

Vertex Pharmaceuticals wins FDA fast track designation for its cell-based diabetes treatment

Vertex Pharmaceuticals announced today that FDA has granted fast track designation for its VX-880, an investigational human stem cell-derived islet cell therapy for type 1 diabetes.

Vertex has begun a clinical trial for VX-880 (formerly known as STx-02) in patients with type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemia awareness.

“Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets. We are very pleased to have received FDA’s fast track designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients,” said Bastiano Sanna, EVP and chief of cell and genetic therapies at Vertex.

Dr. Camillo Ricordi, director of the Diabetes Research Institute (DRI) and the Cell Transplant Center at the University of Miami, said…