Pharmaceutical Technology

MAY 17, 2023

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

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 95

Gene Therapy FDA Approval Gene Clinical Trials 95

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. According to the deal, Sanofi will make an upfront payment of $25m to Scribe.

Development 130

Development In-Vivo Genomics Genome 130

XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Pharmaceutical Technology

SEPTEMBER 14, 2022

As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. The use of engineered immune cytokines that selectively induce the expansion of TILs is being investigated as an alternative to IL-2 stimulation.

Immune Response 246

Immune Response In-Vivo Production Manufacturing 246

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

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. It will also use a variation of CRISPR/Cas9 known as CRISPR/Casx which is smaller, making it easier to deliver using viral vectors for example, and more efficient at modifying genes in the body.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

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.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

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.

Gene Therapy 52

Gene Therapy Gene Development In-Vivo 52

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.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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.

Gene Expression 112

Gene Expression Development RNA Drugs 112

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

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. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

pharmaphorum

APRIL 29, 2021

To achieve this, the agenda will allow delegates to: Explore the translation of BBB-penetrating antibodies to non-human primates , and new possibilities for in vivo brain imaging of antibody neuropharamacokinetics. A Meeting Point for the Field’s Experts. 3-day Conference | June 28-30, 2021. About Hanson Wade.

Drug Delivery 52

Drug Delivery Gene Therapy In-Vivo Antibody 52

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Such challenges highlight a significant unmet need and call for further advancements in the field.

Gene Editing 52

Gene Editing Manufacturing Gene In-Vivo 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

Engineer 52

Engineer Containment In-Vivo Production 52

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. He is currently a professor at the University of Copenhagen where his lab focuses on PNAs in regard to drug discovery, gene targeting, antisense principles, cellular and in vivo delivery and administration of biopharmaceuticals.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Roots Analysis

AUGUST 23, 2024

Applications of mRNA Vaccine and Therapeutics Recent advances using nucleoside-modified mRNA improved protein expression and diminished activation of innate sensors allowing largely overcoming conventional roadblocks concerning mRNA stability, inefficient in vivo delivery and activation of innate sensors by RNA molecules.

Protein 40

Protein Vaccine Vaccination Immune Response 40

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 52

FDA Approval Manufacturing In-Vivo Trials 52

The Pharma Data

OCTOBER 26, 2020

The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system. Key findings of the trial: . – .

Vaccine 40

Vaccine Vaccination Trials Containment 40

XTalks

NOVEMBER 29, 2024

129 Through this acquisition, Roche secures proprietary technologies such as Poseida’s Cas-CLOVER gene editing platform and in-house good manufacturing practice (GMP) manufacturing capabilities. Poseida’s genetic engineering platforms stand out for their precision, safety and potential to open new therapeutic avenues.

Gene Therapy 100

Gene Therapy Gene Gene Editing Genetic Engineering 100