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 A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Pharmaceutical Technology

APRIL 4, 2023

Vertex Pharmaceuticals and CRISPR Therapeutics have completed the rolling submission of biologics licence applications (BLAs) to the US Food and Drug Administration (FDA) for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Gene Therapy 246

Gene Therapy Gene Editing In-Vivo Gene 246

pharmaphorum

FEBRUARY 1, 2024

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, pointing to the potential of CRISPR-based drugs delivered in vivo to treat diseases.

Gene Editing 111

Gene Editing In-Vivo Drugs Clinical Trials 111

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. thalassemia.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.

Gene Editing 97

Gene Editing In-Vivo Gene Sequencing Gene 97

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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Gene Editing Trials Drugs In-Vivo 98

pharmaphorum

NOVEMBER 18, 2022

Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial. LCA10 is caused by mutations in the CEP290 gene and accounts for approximately 20?30% 30% of all LCA cases.

Sales 92

Sales In-Vivo Trials Gene Editing 92

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. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

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. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Gene Therapy Gene FDA Approval In-Vivo 98

World of DTC Marketing

JANUARY 31, 2021

Porter points out that R&D seems to have been replaced with mergers and acquisitions, but she misses the big picture by focusing solely on drug costs. So where to start… Congresswoman Porter may want to look at the percentage of generic drugs available virus branded (89%). In pharma R&D returns have declined to 1.8

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In-Vivo Generic Drugs Drugs Gene Therapy 187

Pharmaceutical Technology

MAY 10, 2023

The research teams will examine the use of CytoMed’s allogeneic gdTc on multiple AML and breast cancer subtypes in vitro and in vivo. Data from the study are expected to support an investigational new drug application with the US Food and Drug Administration for the allogeneic use of gdTc to treat blood and solid cancers.

Gene Therapy 147

Gene Therapy In-Vivo In-Vitro Gene 147

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

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Gene Editing Gene Gene Therapy Drugs 59

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

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Gene Expression Development RNA Drugs 112

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

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Gene Editing Gene In-Vivo Clinical Trials 69

Pharmaceutical Technology

MAY 18, 2023

Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivo genetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We

Gene Therapy 130

Gene Therapy In-Vivo Gene Genetics 130

Pharmaceutical Technology

SEPTEMBER 29, 2022

Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

Pharmaceutical Technology

MAY 24, 2023

to investigational new drug-enabling trials, expected in 2024, to treat aggressive diffuse large B-cell lymphomas (DLBCLs). Morphogenesis is targeting a newly identified delta receptor on myeloid-derived suppressor cells (MDSCs) using its new bi-functional antibody-drug conjugates (ADCs) class.

Gene Therapy 130

Gene Therapy In-Vivo In-Vitro Immune Response 130

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.

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Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

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In-Vivo Gene Editing Gene Therapy Gene 52

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

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Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. MUNICH, Germany, Jan.

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Gene Therapy Gene Development In-Vivo 52

pharmaphorum

FEBRUARY 19, 2021

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

Pharmaceutical Technology

MARCH 9, 2023

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

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In-Vivo Antibody Genetics Gene 130

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

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Gene Editing Genetics Drugs Gene 52

Roots Analysis

FEBRUARY 27, 2024

The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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Genome Genomics Gene In-Vivo 40

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery.

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Gene Therapy Gene Gene Editing In-Vivo 40

Delveinsight

FEBRUARY 11, 2021

Researchers from the University of Pennsylvania’s Abramson Cancer Center contemplate they have discovered one key to the lack of response, and it may be targetable with different drug combinations, as per their announcement. Avrobio gene therapy eradicates toxic substrate in Fabry patient.

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Gene Therapy Gene Immune Response In-Vivo 52

Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system.

Marketing 52

Marketing Gene Drugs Protein 52

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Roots Analysis

AUGUST 8, 2023

Furthermore, a wide variety of drugs and macromolecules, including DNA , proteins, and imaging agents, can be encapsulated in liposomal vesicles due to their unique ability to entrap both lipophilic and hydrophilic substances. The various therapeutic applications of liposomes in drug delivery have been highlighted in the figure.

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Drug Delivery Systems Drug Delivery Drugs In-Vivo 52

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

Roots Analysis

JANUARY 27, 2025

The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases. These therapies have emerged as viable alternatives to traditional treatment options, including small molecule drugs.

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Manufacturing Contamination Production Gene Therapy 40