Clinical Trials, Gene, In-Vivo and Life Science

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

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

In-Vivo Gene Editing Trials Gene

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

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.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

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. 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.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Forbion European Acquisition and enGene sign merger deal

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy

Gene Therapy Gene In-Vivo Life Science

Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

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.

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

The challenges and trends of cell & gene therapies

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

Gene Therapy

Gene Therapy Gene Manufacturing Medicine

Life Science Trends to Look Out for in 2024

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.

Life Science

Life Science Gene Editing Development Clinical Trials

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

Gene Therapy

Gene Therapy Gene Immune Response In-Vivo

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

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.

Clinical Trials

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain

Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion

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

In-Vivo Marketing Engineer Genetics

Strategies to Successfully Manage Complex Cell Therapy Clinical Trials

XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. In a recent webinar, Dr. Vassallo discussed the operational considerations for complex cell therapy clinical trials.

Clinical Trials

Clinical Trials Clinical Trials Trials Gene Therapy

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

XTalks

FEBRUARY 27, 2024

TIL vs CAR T-Cell Therapies While CAR T-cell therapies involve inserting a CAR engineered to target a specific cancer cell protein into a patient’s T cells, TIL-based T-cell therapies like Amtagvi involve separating TILs from a patient’s tumor (in autologous therapies) and expanding them ex vivo.

FDA Approval

FDA Approval Manufacturing In-Vivo Trials

What an innovation centre means for 3D biology

Drug Discovery World

NOVEMBER 16, 2022

In April 2021, life sciences solutions provider Molecular Devices launched its Organoid Innovation Centre (OIC), an initiative designed to help scientists leverage the potential of 3D biology throughout the drug discovery process. . 2D vs 3D . 2D biology has been used by pharmaceutical researchers for decades.

Drugs

Drugs In-Vivo In-Vitro Scientist

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

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

JULY 18, 2023

Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing.

DNA

DNA Genome Genomics RNA

The evolution of assays for immuno-oncology research

Drug Discovery World

FEBRUARY 15, 2023

Over 250 clinical trials are currently in progress, and novel immuno-oncological treatments are already proving to be successful. Cancer is a complex heterogenous multistep disease characterised by uncontrolled cell proliferation combined with a dysregulated immune response. This continues to be the standard of care.

Research

Research In-Vitro In-Vivo Development

Reflecting on PEGS Europe 2023

Drug Discovery World

NOVEMBER 30, 2023

The next presentation attended in this track was the keynote presentation, which was given by Kate Smith, PhD, Head of UK Protein & Cell Sciences at GSK. DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. GSK has developed high-throughput mammalian and E.

Antibody

Antibody Protein Engineer In-Vivo

Clinical Research Informer

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

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

Trending Sources

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

Forbion European Acquisition and enGene sign merger deal

Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

The challenges and trends of cell & gene therapies

Life Science Trends to Look Out for in 2024

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion

Strategies to Successfully Manage Complex Cell Therapy Clinical Trials

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

What an innovation centre means for 3D biology

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

CRISPR breakthroughs: New solutions for common diseases

The evolution of assays for immuno-oncology research

Reflecting on PEGS Europe 2023

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