Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. The BLAs are supported by data obtained from the ongoing Phase III CLIMB-111 and CLIMB-121 trials, along with an ongoing long-term follow-up CLIMB-131 trial. The applications include requests for priority review.

Gene Therapy 246

Gene Therapy Gene Editing In-Vivo Gene 246

BioSpace

APRIL 29, 2024

The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.

Gene Editing 88

Gene Editing Gene Medicine In-Vivo 88

Worldwide Clinical Trials

NOVEMBER 27, 2023

In trials, Casgevy was shown to help prevent episodes of debilitating pain, known as vaso-occlusive crises, that often plague patients affected by sickle cell disease. In June 2023, the U.S. Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

XTalks

APRIL 22, 2022

Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry?

Clinical Trials 98

Clinical Trials Clinical Trials Trials Antibody 98

XTalks

APRIL 14, 2022

Conventional in vitro and in vivo studies are used in the drug development pipeline. Take this quiz to test your knowledge about in silico trials! Register for this free, upcoming webinar to learn more about the benefits of implementing in silico clinical trials with mechanistic models for drug development.

Trials 98

Trials In-Vivo In-Vitro Clinical Trials 98

Pharmaceutical Technology

MAY 16, 2023

The approval also includes clearance for a Phase I clinical trial protocol of NXP900. Nuvectis now has two precision oncology, clinical-stage drug candidates, each with several potential opportunities in cancers that represent serious conditions of unmet medical need. “We

In-Vivo 130

In-Vivo In-Vitro Clinical Trials Clinical Trials 130

Pharmaceutical Technology

JULY 14, 2022

AVR-RD-05 makes use of the own hematopoietic stem cells (HSCs) of a patient that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. Next year, Avrobio is expected to initiate a collaborator-sponsored Phase I/II clinical trial for Hunter syndrome in partnership with the University of Manchester, UK.

Gene Therapy 130

Gene Therapy Gene Drugs In-Vivo 130

Pharmaceutical Technology

OCTOBER 5, 2022

In 2010, two patients with end-stage refractory chronic lymphocytic leukaemia (CLL) were administered Novartis’ autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, Kymriah (tisagenlecleucel), as part of a Phase I trial.

In-Vitro 279

In-Vitro In-Vivo Sales Clinical Trials 279

BioSpace

OCTOBER 18, 2021

On Monday, LogicBio Therapeutics unveiled clinical trial results demonstrating the first-ever in vivo, nuclease-free genome editing in little humans.

In-Vivo 82

In-Vivo Gene Genome Genomics 82

Pharmaceutical Technology

APRIL 28, 2023

The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023. The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023. g/dL and 45.4%

Gene Therapy 264

Gene Therapy Drugs Gene Gene Editing 264

Pharmaceutical Technology

JULY 20, 2022

Roche will also make further payments to Avista during the collaboration’s research phase and clinical, sales-based milestone and royalty payments for resulting products. Avista's computationally steered, in vivo scAAVengr platform uses a high-throughput method with the integrated quantitative validation of new cell-specific AAVs.

Gene Therapy 147

Gene Therapy Gene Development In-Vivo 147

Pharmaceutical Technology

SEPTEMBER 14, 2022

The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.

In-Vivo 130

In-Vivo Clinical Trials Clinical Trials Engineer 130

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

Pharmaceutical Technology

JUNE 21, 2023

It is designed to enhance the function of regulatory T cells (Tregs) in vivo to treat the systemic and neuro-inflammation underlying autoimmune and neurodegenerative diseases. This had a potent neuroprotective effect on nigral dopaminergic neurons in affected mice.

In-Vivo 130

In-Vivo Immune Response Clinical Trials Clinical Trials 130

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

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

FEBRUARY 1, 2023

Ellipses Pharma stated that the dual FLT3 inhibition and Aurora kinase demonstrated to overcome the acquired resistance to selectively inhibit FLT3 in vitro and in vivo. According to the company, approximately one-third of AML patients are diagnosed with FLT3 mutations related to a higher relapse risk and poor clinical outcomes.

FDA Approval 130

FDA Approval In-Vivo In-Vitro Trials 130

Camargo

NOVEMBER 11, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Unpacking the (Black) Box: Antares Licenses Urology Product with Boxed Warning. of hyponatremia, or low blood sodium levels. In October, the U.S.

Development 242

Development Bioequivalency Generic Drugs Production 242

Pharmaceutical Technology

FEBRUARY 13, 2023

The continued high level of interest in small molecules presents multiple opportunities to select a candidate that is ‘developable’, with subsequent rapid progression toward first-in-human (FIH) clinical testing. With all this in mind, how do drug developers know which strategy and approach is right for their molecule at this early stage?

Development 130

Development In-Vivo Drugs Clinical Trials 130

World of DTC Marketing

JANUARY 31, 2021

I have been recruited by these companies who promise huge payouts when they are acquired if their drug shows promise in clinical trials. Ex vivo (where cells are genetically modified outside the body) cell and gene therapies have generated considerable excitement on their potential to cure previously incurable diseases.

In-Vivo 187

In-Vivo Generic Drugs Drugs Gene Therapy 187

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 52

Clinical Trials Clinical Trials Trials Gene Therapy 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.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

Pharmaceutical Technology

MAY 18, 2023

Other investors include Investissement Québec, Omega Funds, Cowen Healthcare Investments, CTI Life Sciences Fund III, Northleaf Capital Partners, BVF Partners and Vivo Capital. The combined company will be named enGene Holdings and its common shares are expected to be listed on the Nasdaq.

Gene Therapy 130

Gene Therapy Gene In-Vivo Life Science 130

pharmaphorum

JANUARY 13, 2021

IO Biotech, an oncology specialist formed and backed by Denmark’s Novo Holdings, has raised €127 million ($155 million) to further develop its cancer vaccine technology that has boosted efficacy of PD-1 immunotherapy in early trials. The post IO Biotech raises $155m to develop breakthrough cancer vaccine appeared first on.

Vaccine 95

Vaccine Vaccination Development In-Vivo 95

Camargo

APRIL 28, 2021

Conducting a clinical study in a pediatric patient population requires careful planning, as providing a benefit to a child without causing harm is a weighty responsibility. First Steps Toward Initiating Pediatric Clinical Studies. Data Collection from Preclinical and Adult Studies.

Clinical Trials 130

Clinical Trials Clinical Trials In-Vivo Drugs 130

XTalks

DECEMBER 13, 2024

The DREAMM-7 Phase III trial has spotlighted the potential of Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BVd) to improve survival outcomes for relapsed or refractory multiple myeloma. DREAMM-7, an international, open-label trial, enrolled 494 participants with at least one prior multiple myeloma therapy.

Antibody 99

Antibody In-Vivo Trials In-Vitro 99

BioTech 365

SEPTEMBER 29, 2020

First patient was administered today The trial aims at validating the safety of continuous administration of Ivermectin in oral form Simultaneously, several long-acting injectable formulations based on MedinCell’s BEPO® technology are being tested in vivo A 1-month active injectable prophylactic … Continue reading →

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials Trials 40

pharmaphorum

JULY 22, 2021

We have completed phase 2 trial of a multi-antibody drug for influenza, CT-P27, and since 2015 we have been developing a therapeutic antibody to treat the Middle East Respiratory Syndrome (MERS), CT-P38. In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19.

Antibody 105

Antibody Clinical Trials Clinical Trials Trials 105

XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

FEBRUARY 15, 2023

The pharmaceutical industry continues to be a hotbed of innovation, with activity driven by the evolution of new treatment paradigms, and the gravity of unmet needs, as well as the growing importance of technologies such as pharmacogenomics, digital therapeutics, and artificial intelligence.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

pharmaphorum

FEBRUARY 19, 2021

US biotech Excision BioTherapeutics has raised $60 million to test a potential HIV cure in the clinic, which would use CRISPR technology to snip out the viral code from human cells and tissues. We have proven the technology and candidate programs in vitro and in vivo in both small animal models and primate models.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

FEBRUARY 13, 2022

Quris’ BioAI safety prediction platform – which is based on human tissue samples on chips, nanosensors and machine learning – will be compared to traditional in vitro and in vivo laboratory techniques for spotting toxicity. The post Merck will assess Quris’ AI ‘patient-on-a-chip’ drug safety appeared first on.

Drugs 98

Drugs In-Vivo In-Vitro Pharma Companies 98

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

BioTech 365

SEPTEMBER 17, 2020

Positive results for Step-1 of Phase 3 clinical trial of Tedopi® in non-small cell lung cancer Positive preclinical and in human ex vivo results with CoVepiT vaccine against COVID-19; clinical entry expected by the end of 2020 or early 2021 … Continue reading →

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials Vaccination 40

XTalks

AUGUST 19, 2022

XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research. Register for this free webinar to learn why the agile clinical trial model is a great fit for many rare disease categories, how to navigate the regulatory landscape for agile clinical trials in rare diseases, how to design studies and more.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95