FDA Approval Protein Expression Therapies Treatment 
Drug Discovery World
JUNE 23, 2023
Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.
Drug Discovery World
SEPTEMBER 25, 2024
Enhertu is now one of 13 ADCs approved by the FDA, with hundreds of others in development, some of which have been snapped up by big pharma in substantial deals, as is evident in the figures below. And, last but not least, therapy with ADCs offers physicians the decisive advantage that it can be administered on site at the hospital.
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Advarra
JANUARY 11, 2024
The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.
The Pharma Data
JANUARY 15, 2021
hereafter, Daiichi Sankyo) and AstraZeneca’s Enhertu ( fam-trastuzumab deruxtecan -nxki) has been approved in the U.S. for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. In the U.S.,
The Pharma Data
SEPTEMBER 28, 2021
The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to 6 weeks aged and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne dystrophy (DMD).
The Pharma Data
DECEMBER 8, 2020
Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021.
Conversations in Drug Development Trends
MAY 30, 2024
Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. Initial approval in 2022: FDA, U.S.
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