FDA Approval, Gene, Gene Therapy and In-Vivo

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 FDA Approval Gene 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

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

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

This week in drug discovery (18-22 March)

Drug Discovery World

MARCH 22, 2024

This has been an interesting week for cell and gene therapies, with two landmark FDA approvals, two significant fundraising efforts and potentially ground-breaking study results in glioblastoma. News round-up for 18-22 March by DDW Digital Content Editor Diana Spencer.

Gene Therapy

Gene Therapy In-Vivo FDA Approval Drugs

This week in drug discovery (19-23 February)

Drug Discovery World

FEBRUARY 23, 2024

In a breakthrough for advanced therapies, this week saw the FDA approve the first ever cell therapy for solid tumour cancers, but there were other significant developments in the cell and gene therapy space. News round-up for 19-23 February by DDW Digital Content Editor Diana Spencer.

Gene Therapy

Gene Therapy Insulin FDA Approval Drugs

FDA approves Phase III cerebral Adrenoleukodystrophy trial

Drug Discovery World

JUNE 12, 2023

Radiological changes were similar to those attained with Hematopoietic Stem Cell Transplant (HSCT) or ex-vivo gene therapy. ” The post FDA approves Phase III cerebral Adrenoleukodystrophy trial appeared first on Drug Discovery World (DDW).

FDA Approval

FDA Approval Trials Radiology In-Vivo

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

Overcoming diverging regulatory expectations to bring CGTs to market

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy

Gene Therapy Marketing Gene DNA

FDA approves first in vivo gene insertion programme in infants

Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

In-Vivo

In-Vivo FDA Approval Gene Gene Editing

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

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

Drug discovery quarterly review: Second quarter 2023

Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

Drugs

Drugs Vaccination Vaccine Medicine

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

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 Manufacturing Protein Bacteria

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 Genetics

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

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

Drugs

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Clinical Research Informer

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

FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy

Trending Sources

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

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

This week in drug discovery (18-22 March)

This week in drug discovery (19-23 February)

FDA approves Phase III cerebral Adrenoleukodystrophy trial

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

Overcoming diverging regulatory expectations to bring CGTs to market

FDA approves first in vivo gene insertion programme in infants

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

Life Science Trends to Look Out for in 2024

Drug discovery quarterly review: Second quarter 2023

FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

Biologics – The Next Step in Revolutionary Medication

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

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

Advances in neuroscience drug discovery

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