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.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

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

Pharmaceutical Technology

MAY 18, 2023

It also demonstrates KSQ’s ability to deliver results – our original collaboration, initiated in 2021 and focused on the discovery of novel NK cell and T cell-based targets, has already achieved several research milestones.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 130

Gene Therapy In-Vivo Gene Genetics 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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Development 130

Development In-Vivo Genome Genomics 130

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

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Gene Editing Gene Gene Therapy In-Vivo 145

Delveinsight

FEBRUARY 11, 2021

Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

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

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). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Gene Editing In-Vivo Gene Sequencing Gene 97

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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Genome Genomics Gene In-Vivo 40

The Pharma Data

NOVEMBER 22, 2020

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years’ experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. MILAN, Italy and NEW YORK, Nov. About Genenta Science.

Gene Therapy 40

Gene Therapy Gene In-Vivo Clinical Trials 40

Pharmaceutical Technology

JULY 20, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes.

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Gene Therapy Gene Development In-Vivo 147

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. Bluebird spins off to two companies, cleaving off its gene therapy, and cancer units. Further, Birinapant also complements the anti-tumor activity of the immune system.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

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 Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 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

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 79

Gene Therapy FDA Approval Gene Clinical Trials 79

Pharmaceutical Technology

AUGUST 18, 2022

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. An ex-vivo lentiviral vector (LVV) gene therapy, Zynteglo’s every dose is made by genetically modifying the bone marrow stem cells of the patient to produce functional beta-globin.

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Gene Therapy Gene Genotype In-Vivo 246

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

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.

Manufacturing 40

Manufacturing Production Contamination Gene Therapy 40

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.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Pharmaceutical Technology

MAY 10, 2023

CytoMed Therapeutics has signed a research collaboration deal with the University of Texas MD Anderson Cancer Center (MD Anderson) in the US to use gamma-delta T cells (gdTc) to treat acute myeloid leukaemia (AML) and breast cancer. MD Anderson leukaemia associate professor Venkata Lokesh Battula will lead the research study.

Gene Therapy 147

Gene Therapy In-Vivo In-Vitro Gene 147

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

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

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

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

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.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

XTalks

DECEMBER 20, 2023

AI and Machine Learning: Transforming Research Artificial intelligence (AI) and machine learning (ML) are revolutionizing research approaches in the life sciences. Below are some of the key trends that will be shaping the future of the life sciences in 2024 and beyond.

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Life Science Gene Editing Development Clinical Trials 119

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.

Gene Editing 52

Gene Editing Manufacturing Gene In-Vivo 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 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. There are many notable differences between a Contract Research Organization (CRO) and a Contract Vivarium (CV). Photo courtesy of Mispro.

Marketing 52

Marketing In-Vivo Scientist Research 52

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.

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

SEPTEMBER 18, 2023

Even though, appropriately preserved biologics have immense potential for use in basic research and other medical applications, damage sustained by the membrane during freezing and thawing results in osmotic shock, which can ultimately lead to cell death.

Engineer 52

Engineer Containment In-Vivo Production 52

The Pharma Data

DECEMBER 11, 2020

of Children’s Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues found that gene editing using CRISPR-Cas9 is safe and feasible and can provide clinical benefit to patients with sickle cell disease and transfusion-dependent ß-thalassemia. In one study, Haydar Frangoul, M.D., Abstract No.

Gene Editing 52

Gene Editing Gene Gene Therapy In-Vivo 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.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 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 Vaccination Vaccine Medicine 52