FDA Approval, Gene and In-Vivo - Clinical Research Informer

FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

Pharmaceutical Technology

SEPTEMBER 19, 2022

The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio ’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. The treatment is indicated for usage in boys aged four to 17 years with CALD.

FDA Approval

FDA Approval In-Vivo Gene Therapy Clinical Trials

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy

Gene Therapy Gene Genotype In-Vivo

Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Zynteglo’s hefty price tag of $2.8

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

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Bridging Innovation & Patient Care: The Growing Role of AI

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

Gene Therapy

Gene Therapy Gene FDA Approval In-Vivo

FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy

BioSpace

JUNE 21, 2023

Elevidys, authorized Thursday to treat ambulatory patients 4 to 5 years of age, is the first in vivo gene therapy to win the FDA’s accelerated approval. It is also the first such therapy for DMD.

Gene Therapy

Gene Therapy Gene FDA Approval In-Vivo

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing

Gene Editing Gene In-Vivo Clinical Trials

Life Science Trends to Look Out for in 2024

XTalks

DECEMBER 20, 2023

In 2023, Dr. Varshney notes that the US Food and Drug Administration (FDA) issued a request for industry comment on the integration of AI in drug and biologics development. CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason.

Life Science

Life Science Gene Editing Development Clinical Trials

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

XTalks

FEBRUARY 27, 2024

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

FDA Approval

FDA Approval Manufacturing In-Vivo Trials

Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

MAY 28, 2024

Today there are six CAR-T products with FDA approval for the treatment of multiple myeloma and B cell malignancies, with many related and next-generation products under development. CAR-T products with FDA approval for treatment of B cell lymphomas express receptors engineered to recognize CD19.

In-Vivo

In-Vivo Clinical Trials Clinical Trials FDA Approval

Biologics – The Next Step in Revolutionary Medication

Roots Analysis

JANUARY 14, 2024

Given the ongoing scientific advancements and the rise of FDA-approved biologics, the pharmaceutical industry seems to be approaching the era of biologics. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

Contract Manufacturing

Contract Manufacturing Protein Manufacturing Production

What the Glycome Can Tell Us About Persistent HIV Infection

XTalks

AUGUST 12, 2020

Related: FDA Approves Rukobia for HIV Patients with Limited Treatment Options. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

RNA

RNA Protein DNA In-Vivo

Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

Sales

Sales Vaccination Vaccine Medicine

Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

The Pharma Data

OCTOBER 26, 2020

The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system. Key findings of the trial: . – .

Vaccination

Vaccination Vaccine Trials Containment

Merus’ Bizengri Gets FDA Accelerated Approval for NSCLC and Pancreatic Adenocarcinoma with NRG1 Gene Fusions

XTalks

DECEMBER 6, 2024

The US Food and Drug Administration (FDA) has granted accelerated approval to Merus Bizengri (zenocutuzumab-zbco) for the treatment of metastatic non-small cell lung cancer (NSCLC) and advanced pancreatic adenocarcinoma with NRG1 gene fusions in patients who have had disease progression during or after prior systemic therapy.

Gene

Gene In-Vivo In-Vitro Genetics

Clinical Research Informer

FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Webinars

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Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

Webinars

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

Life Science Trends to Look Out for in 2024

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

Biologics – The Next Step in Revolutionary Medication

What the Glycome Can Tell Us About Persistent HIV Infection

Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

Merus’ Bizengri Gets FDA Accelerated Approval for NSCLC and Pancreatic Adenocarcinoma with NRG1 Gene Fusions

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