Remove FDA Approval Remove Immune Response Remove Virus
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Sarepta Strengthens ELEVIDYS Safety Measures for Non-Ambulatory Duchenne Patients

The Pharma Data

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Sarepta’s team is exploring adding sirolimus — a well-established immunosuppressive medication — to the regimen in a way that might enable a greater degree of control over the patient’s immune response.

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A Visual Guide to Gene Delivery

Codon

Casgevy , the first FDA-approved CRISPR-based therapy, is also an ex vivo gene therapy; it aims to cure sickle cell disease by editing a patient’s blood-producing stem cells so they generate functional fetal hemoglobin, then returning these corrected cells to the bloodstream. This changed with the discovery of AAVs.

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A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays.

DNA
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Clover Biopharmaceuticals Announces Positive Phase 1 Data for its Adjuvanted S-Trimer COVID-19 Vaccine Candidates

The Pharma Data

Strong Th1 cell-mediated immune responses were also observed for the vaccine candidates with either adjuvant. We are encouraged by the high level of neutralizing antibodies in combination with the strong Th1 response which we believe could play an important role in controlling infection.

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PFIZER AND BIONTECH ANNOUNCE SUBMISSION OF INITIAL DATA TO U.S. FDA TO SUPPORT BOOSTER DOSE OF COVID-19 VACCINE

The Pharma Data

Food and Drug Administration (FDA) to support the evaluation of a third, or booster, dose of the companies’ COVID-19 vaccine (BNT162b2) for future licensure. We are pleased to submit these data to the FDA as we continue working together to address the evolving challenges of this pandemic.”. “We CEO and Co-founder of BioNTech.

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ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates

The Pharma Data

In the study, immunization with the hAd5-COVID-19 vaccine inhibited SARS-CoV-2 virus replication in 100% (10 of 10) of Rhesus macaques, with a drop in viral replication starting on the first day of vaccine administration, and undetectable viral levels as early as three to five days post-challenge in most of the animals.

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Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

The Pharma Data

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older.