Remove 2016 Remove Packaging Remove Regulations
article thumbnail

Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

s, are built with base editing technology, a gene editing technique developed by Liu’s team in 2016 to directly convert an individual DNA base pair into a different base pair. The team that treated K.J. was led by physician-scientists Kiran Musunuru and Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia and U.

Treatment 133
article thumbnail

A Visual Guide to Genome Editors

Codon

pyogenes protein — whose compactness makes them far easier to package into viral vectors and deliver into the human body. For example, one can fuse a deactivated Cas9 protein to so-called “transcriptional regulators” to either activate or repress genes without directly editing the genome at all.

DNA 83
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

FDA Issues Long-Awaited QMSR Final Rule

FDA Law Blog: Biosimilars

By Véronique Li, Senior Medical Device Regulation Expert & Ana Loloei & Allyson B. Mullen — More than five years after FDA first announced its plan to harmonize 21 CFR Part 820 with ISO 13485, on February 2, 2024, FDA finally issued the Quality Management System Regulation (QMSR) Final Rule. The new § 820.10

FDA 59
article thumbnail

Analysis Chemical Thank You PFAS regulation in California (late 2023 edition)

Agency IQ

PFAS regulation in California (late 2023 edition) California consistently maintains its status as both one of the most important economies in the world as well as one of the most regulated states in the United States. In 2019, the state began to significantly ramp up its PFAS regulation and research.

article thumbnail

Transitioning to eCTD v4.0

The Premier Consulting Blog

Starting in 2016, many versions were developed through internal collaboration and change requests until, in 2022, eCTD v4.0 Sponsors should prepare by reviewing implementation packages from the ICH and applicable regions. Implementation Package v1.5 Implementation Package v1.5 Module 1 Implementation Package FDA eCTD v4.0

article thumbnail

CMS Proposals Would Raise the Bar on Bona Fide Service Fees for Average Sales Price

FDA Law Blog: Biosimilars

Kirschenbaum — The Calendar Year 2026 Medicare Physician Fee Schedule (PFS) proposed rule ( here ), which was issued yesterday by CMS, contained important amendments to the regulations on Medicare Part B average sales price (ASP) reporting. The payment limit for most Part B drugs is ASP plus 6 percent. with certain exceptions.

article thumbnail

Article EMA Thank You New MHRA Windsor Framework documents guide post-Brexit flow of medicines for U.K., Great Britain, and the E.U.

Agency IQ

government was put in place on February 27, 2023 and includes a number of proposed regulations to address trade relations between the E.U. First: all medicines for Northern Ireland must be approved by the MHRA The new regulation (EU) 2023/1182 applies to medicines that require marketing authorization. and the U.K.