FDA Approval Protein Expression Therapies Trials 
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.
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.
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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. Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. Nuance Pharma .
The Pharma Data
JANUARY 15, 2021
“This approval represents the first time a HER2 directed medicine has demonstrated a significant improvement in survival compared to chemotherapy for patients following initial treatment in the metastatic setting and it has the potential to become the new standard of care for this patient population.”. Regular approval by the U.S.
Conversations in Drug Development Trends
MAY 30, 2024
Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Initial approval in 1994: FDA, U.S.
Alta Sciences
APRIL 17, 2024
Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.
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