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

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 98

Gene Therapy FDA Approval Gene In-Vivo 98

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

Gene Therapy 52

Gene Therapy FDA Approval Gene Gene Editing 52

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.

Gene Editing 60

Gene Editing Licensing Licensing FDA Approval 60

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

Gene Therapy 52

Gene Therapy Gene FDA Approval Clinical Trials 52

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. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 118

Research Gene Editing Gene Genetics 118

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

Gene Therapy 57

Gene Therapy Gene Antibody FDA Approval 57

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.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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.

Marketing 130

Marketing Gene Editing Production Sales 130

Drug Discovery World

APRIL 4, 2023

Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

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

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

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

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

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 Vaccination Vaccine Gene 111

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. 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

Drug Discovery World

FEBRUARY 22, 2024

The FDA has approved the supplemental Biologics License Application (sBLA) supporting the traditional approval of LEQEMBI (lecanemab-irmb) injection, for reducing the rate of disease progression and to slow cognitive and functional decline in adults with Alzheimer’s disease (AD).

Drugs 57

Drugs Gene Clinical Trials Clinical Trials 57

Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome.

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Research Gene Gene Therapy Genome 264

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

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

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 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 52

Manufacturing Production Development Clinical Trials 52

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.

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RNA Vaccination Vaccine Pharma Companies 111

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.

Antibody 52

Antibody Immune Response Production Engineer 52