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 59

Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. The move marks a continuation in its ongoing commitment to exploring gene-editing technology. Novartis also recently inked an up-to $1.5

Gene Editing 52

Gene Editing Gene Gene Therapy Genetics 52

XTalks

DECEMBER 13, 2023

Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

Pharmaceutical Commerce

JANUARY 17, 2024

The list price of Casgevy for transfusion-dependent beta thalassemia has yet to be released, however, the cost for the treatment in sickle cell disease is $2.2 million.

Gene Editing 52

Gene Editing FDA Approval Gene 52

BioSpace

DECEMBER 12, 2023

The deal follows the FDA approval of Vertex’s gene-editing sickle cell treatment and Editas’ earlier legal battle over rights to the technology.

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Gene Editing Licensing Licensing FDA Approval 57

Pharmaceutical Technology

FEBRUARY 13, 2023

This is particularly the case for pediatric forms of epilepsy where gene editing techniques like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are becoming more important, says Ferraro. GW Pharmaceuticals’ Epidiolex (cannabidiol), the first-FDA approved CBD treatment , is also a valuable addition, he adds.

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Research Gene Gene Therapy Genomics 264

Pharmaceutical Technology

JANUARY 19, 2023

However, it is possible to target CD33 in the clinic, as evidenced by the FDA approval of Pfizer’s anti-CD33 antibody-drug conjugate Mylotarg (gemtuzumab ozogamicin) in 2000. The first clinical attempt at managing AML with CAR-T cells was reported in 2013 with a small Phase I study that did not demonstrate activity.

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Marketing Gene Editing Production Sales 130

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. As of October 19, 2023, there were 692 FDA-approved devices linked to AI/ML , underscoring the expanding role of these technologies in healthcare.

Research 118

Research Gene Editing Gene Genetics 118

XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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FDA Approval Gene Therapy Gene Clinical Trials 111

pharmaphorum

JULY 13, 2021

At the moment, the only FDA-approved treatments for ATTR cardiomyopathy are Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), which were given a green light by the agency two years ago. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body.

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

XTalks

JANUARY 4, 2024

Gene therapy approvals for rare blood disorders like sickle cell disease and beta thalassemia have been gaining traction lately, with two significant approvals in sickle cell disease recently — CRISPR Therapeutics and Vertex Pharmaceuticals’ CRISPR gene editing drug Casgevy (exa-cel) and bluebird bio’s viral vector therapy Lyfgenia (lovo-cel).

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

Worldwide Clinical Trials

MAY 30, 2024

From drugs previously FDA-approved for gallstone diseases, Parkinson’s disease, and even cancer, researchers are working tirelessly to test and establish whether these pharmacotherapies with well-defined safety profiles may have any potential for efficacy in treating ALS.

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Gene Clinical Trials Clinical Trials Gene Therapy 204

pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies.

RNA 111

RNA Vaccine Vaccination Pharma Companies 111

XTalks

SEPTEMBER 4, 2024

The HELIOS-B results build on earlier success from the HELIOS-A trial, which led to the US Food and Drug Administration (FDA) approval of vutrisiran (Amvuttra) for polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR).

Trials 52

Trials RNA Protein Gene Editing 52

XTalks

MARCH 28, 2024

Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. For example, gene editing can possibly be used to correct cancer-causing mutations or enhance the effectiveness of chimeric antigen receptor (CAR) T-cell therapy.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

AUGUST 2, 2023

Paxlovid was first authorized under the FDA Emergency Use Authorization in December 2021 ; however, it has received FDA approval on May 25, 2023. pneumoniae serotypes) replaced the company’s first pneumococcal conjugate vaccine Prevnar (PCV7, approved by the FDA in February 2000) in a February 2010 FDA approval.

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Sales Manufacturing FDA Approval Life Science 98

WCG Clinical

OCTOBER 21, 2024

Advances in research, such as gene editing and cellular therapies, have opened new treatment possibilities for both conditions. Recent FDA approvals of gene therapies for sickle cell and cellular therapies for melanoma have given patients new hope, but these therapies are not without limitations.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Clinical Research 52

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. In 2021, we saw a number of innovations in fields like neuromodulation, with several neurostimulation devices having received FDA approvals.

Life Science 98

Life Science RNA Vaccination Vaccine 98

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

NOVEMBER 2, 2021

Clinical interest in bsAbs gained momentum following the promising data and market success of blinatumomab (BLINCYTO®, Amgen), a fragment-based bispecific T cell engager (BiTE), which first received FDA approval in 2014 and EMA approval in 2015.

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Antibody Immune Response Production Engineer 52

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

DECEMBER 27, 2020

Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies. From these cells we apply proprietary methods to manufacture pure populations of only the cell types which we wish to use in patients.

Manufacturing 40

Manufacturing Production Development Clinical Trials 40

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccine Vaccination Gene 111

XTalks

NOVEMBER 7, 2024

Casgevy: A Game-Changer for Beta Thalassemia CRISPR Therapeutics and Vertex are transforming blood disorder treatment with their gene-editing solution Casgevy (exagamglogene autotemcel [exa-cel]). This one-time gene therapy tackles transfusion-dependent beta-thalassemia (TDT), a disorder causing severe anemia.

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Gene Therapy Gene Editing Gene FDA Approval 105

XTalks

JANUARY 6, 2025

Notably, the ASO drug Vitravene (fomivirsen) received US Food and Drug Administration (FDA) approval for treating cytomegalovirus (CMV) retinitis in AIDS patients. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111