BioCentury
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Mda

BioCentury | Jun 28, 2024
Regulation

Approval of Sarepta DMD gene therapy revives questions on FDA interactions with companies, patient advocates

CBER Director Marks met with biopharma companies, industry-funded advocates while considering gene therapy application
BioCentury | Mar 9, 2024
Discovery & Translation

Science spotlight: Elixirgen’s full-length dystrophin gene therapy, Insilico’s TNIK inhibitor and more

BioCentury’s roundup of translational innovations
BioCentury | Jun 16, 2023
Product Development

Patient organizations take on gene therapy’s redosing problem

If successful, a clinical trial funded by CureDuchenne, MDA and PPMD could have industry-wide consequences for gene therapy
BioCentury | May 1, 2023
Deals

Maze-Sanofi deal in Pompe shows how early clinical data can drive pharma deals

Pharma extends rare disease franchise, paying well-funded biotech $150M in cash and equity investment to embrace substrate reduction approach
BioCentury | Jul 2, 2021
Distillery Therapeutics

Targeting SDCBP in metastatic breast cancer

BioCentury | Apr 16, 2021
Politics, Policy & Law

Pandemic pain: how COVID-19 has devastated patient groups

Patient groups that are an essential part of the life sciences ecosystem are suffering from increased demands and decreased funding
BioCentury | Apr 20, 2018
Company News

Target ALS and partners launch biomarker study

BioCentury | Apr 19, 2018
Company News

Target ALS, partners launch biomarker study

BioCentury | May 26, 2014
Clinical News

Aldeyra Therapeutics preclinical data

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