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

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene Editing Gene Gene Therapy In-Vivo 189

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. It also affects Hispanic Americans, but at a lower prevalence.

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Gene Therapy Gene FDA Approval In-Vivo 98

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company will also receive tiered royalties on the worldwide net sales of products developed from the collaboration.

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Gene Editing Gene In-Vivo Gene Therapy 147

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.

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Gene Therapy Gene In-Vivo Life Science 130

Pharmaceutical Technology

AUGUST 17, 2022

These programmes will include therapies and vaccines in infectious disease and oncology areas. oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. .

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RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

MAY 18, 2023

KSQ will receive future payments of up to $510m on achieving all milestones during the agreement period, along with potential net sales royalties on any commercial product that is the result of the partnership. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy In-Vivo Gene Genetics 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.

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Gene Therapy Gene Development In-Vivo 52

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.

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Gene Editing Gene Gene Therapy In-Vivo 130

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Sanofi will also make tiered royalty payments on net sales of products resulting from the collaboration in the future.

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Development In-Vivo Genomics Genome 130

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

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.

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In-Vivo Gene Editing Trials Gene 52

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

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Gene Therapy Gene In-Vivo Genetics 40

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. The major steps involved in cell therapy manufacturing have been briefly outlined below.

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Manufacturing Contamination Production Gene Therapy 40

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

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Clinical Trials Clinical Trials Gene Therapy Gene 52

pharmaphorum

MAY 27, 2021

Life sciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Funds will be used to expand the company’s presence across Asia and create an innovation hub in Boston for cell and gene therapy technologies.

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Life Science In-Vivo Manufacturing Gene Therapy 59

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

Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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Genomics Genome Gene In-Vivo 40

pharmaphorum

NOVEMBER 4, 2022

” At Novartis, she was involved with projects including multiple sclerosis therapy Kesimpta (ofatumumab), migraine therapy Aimovig (erenumab), and Parkinson’s disease, and the acquisition by Novartis of optogenetic gene therapy company Vedere Bio. .

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In-Vivo Gene Therapy Doctors Doctor 59

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. The rapidly expanding landscape of cell therapy trials has put an operational strain on many clinical sites due to limited resources and infrastructure. “In

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Clinical Trials Clinical Trials Trials Gene Therapy 52

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Besides, BeiGene has an option to co-detail the product in North America, which will be funded in part by Novartis.

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Licensing Licensing Antibody Gene Therapy 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.

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Gene Gene Therapy Gene Expression Regulation 40

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. billion product if it gets approved for both indications.

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Gene Editing Gene In-Vivo Genetics 52

WCG Clinical

MAY 28, 2024

Today there are six CAR-T products with FDA approval for the treatment of multiple myeloma and B cell malignancies, with many related and next-generation products under development. In the context of anticancer treatment, B cell aplasia is considered an undesirable but manageable side effect of these therapies.

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In-Vivo Clinical Trials Clinical Trials FDA Approval 40

The Pharma Data

DECEMBER 13, 2020

Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.

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Business Development Development Gene Editing In-Vivo 40

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

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.

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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.

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In-Vivo In-Vitro Antibody Development 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. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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Gene Editing Manufacturing Gene In-Vivo 52

Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

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Manufacturing Production Contamination Gene Therapy 40

Delveinsight

JANUARY 18, 2021

Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father. GeneTx and Ultragenyx are co-developing the therapy.

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Marketing Gene Drugs Protein 52

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

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Vaccination Vaccine In-Vivo Genetics 52

Roots Analysis

JANUARY 14, 2024

Moreover, the exuberant development of biologics has revolutionized the treatment of a range of therapeutic conditions, which has further contributed to the exponential growth in the current demand for biologic therapies developed by biologics manufacturing companies. Like all drugs, biologics are regulated by the FDA.

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Contract Manufacturing Protein Manufacturing Production 40

The Pharma Data

NOVEMBER 10, 2020

This model has the benefit of giving companies direct oversight of their in vivo studies, allowing them to move faster while operationalizing every dollar of their seed financing. Photo courtesy of Mispro. There’s an emerging business model in biotech town, and it’s called the Contract Vivarium (CV).

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Marketing In-Vivo Scientist Research 52

The Pharma Data

OCTOBER 15, 2020

The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. BeyondSpring – BeyondSpring Inc. Additionally, Parag V. At Spark, he served as head of U.S.

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In-Vivo Business Development Manufacturing Development 52

XTalks

FEBRUARY 27, 2024

Since the activity of TILs can be suppressed by tumor cells, TIL therapies are designed to bolster the number of TILs. Related: FDA Issues Letters to CAR T-Cell Therapy Makers to Include Boxed Warning Amtagvi was approved through the FDA’s Accelerated Approval pathway. TILs are immune cells that attack and penetrate tumors.

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FDA Approval Manufacturing In-Vivo Trials 52

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

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Sales Vaccine Vaccination Medicine 52