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.

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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.

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Drug Discovery World

MAY 9, 2023

The recent passage of the FDA Modernization Act, which removed the requirement for animal testing when suitable alternatives are available, has highlighted the inadequacy of animal models most often used for in vivo research. Fewer than 5% of oncology drugs that enter clinical trials in the US receive US FDA approval.

Development 52

Development Drugs In-Vivo Clinical Trials 52

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).

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Gene Editing Gene Gene Therapy In-Vivo 145

The Pharma Data

JANUARY 17, 2021

Joe Dupere, CEO of Ixaka, commented: “Ixaka’s broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies. A total of $15.4 A total of $15.4 References 1.

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Gene Therapy Gene In-Vivo Genetics 40

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

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Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

SEPTEMBER 28, 2022

It involves an ‘ex vivo’ use of gene-editing, with the technology is used to modify a patient’s own cells outside the body to make foetal haemoglobin (HbF), which can serve as a substitute to regular haemoglobin in both SCD and thalassaemia.

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Gene Editing Gene In-Vivo Clinical Trials 59

The Pharma Data

MAY 3, 2021

DARS also greatly expanded its clinical trials operations, running three clinical trials related to streamlining biosimilars development and studying safety questions related to opioids and over-the-counter drugs,” Strauss wrote. Posted 03 May 2021 | By Michael Mezher . 2020 was a challenging year.

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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.

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Drug Discovery World

APRIL 4, 2023

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment Drug candidate for TUBB4a leukodystrophy, SynaptixBio The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

pharmaphorum

OCTOBER 6, 2022

Thirty years on, GWI has proven itself to be a challenging clinical condition; chronic and multisymptomatic by nature, the disease affects around a third of all veterans who served during the Gulf War. Disease modelling Gulf War Illness. In the mid-to-late 1990s, an unexplained illness began to emerge in army bases across the US.

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In-Vivo Research Genetics Drugs 98

Drug Discovery World

AUGUST 14, 2023

For example, the FDA’s Center for Biologics Evaluation and Research (CBER) 3 , which regulates biological products for human use, differentiates between in vivo gene therapy (new genes are inserted into the body to target cells) and ex vivo gene therapy (cells are modified outside the body and then administered back into the patient).

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Gene Therapy Marketing Gene DNA 52

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

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.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods? Not yet, according to Ewart.

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Research Gene Therapy In-Vivo In-Vitro 72

pharmaphorum

OCTOBER 8, 2020

Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.

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Gene Editing Gene In-Vivo Clinical Trials 60

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. They also wondered whether more pre-clinical data would even be useful as the ultimate test is how the treatment will fare in patients. Vertex and CRISPR Therapeutics are also seeking approval for exa-cel in beta thalassemia, the decision for which is set for March 30, 2024.

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Gene Therapy Gene Gene Editing In-Vivo 59

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 52

Drug Discovery World

FEBRUARY 6, 2023

This is part of why patients in trials and the clinic can experience severe toxicity and adverse events, reject their therapy with an immune response, or completely fail to respond to treatment. DDW’s Megan Thomas caught up with Wyatt McDonnell, CEO and co-founder of Infinimmune. This has consequences for safety and efficacy.

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Antibody Immune Response Genome Genomics 52

Drug Discovery World

JUNE 30, 2023

Through the use of human induced pluripotent stem cells (iPSCs) derived cells, we can improve translation and reduce risk of clinical failure. Through the use of human induced pluripotent stem cells (iPSCs) derived cells, we can improve translation and reduce risk of clinical failure.

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Development Drugs In-Vitro Gene Therapy 52

Drug Discovery World

JULY 22, 2022

For example, in randomised clinical trials of patients with non-small cell lung cancer (NSCLC), pembrolizumab has been shown to be effective in slowing down disease progression and improving survival rates 1. demonstrated the ability to model clinical responses to ACT in autologous HIS mice 4. Jespersen et al.

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In-Vivo Engineer In-Vitro Antibody 52

Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate.

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Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

APRIL 8, 2022

Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19. Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19.

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Immune Response In-Vivo Genetic Analysis Research 52

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). 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.

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Gene Therapy FDA Approval Gene In-Vivo 98

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. 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.

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Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

JULY 5, 2023

It’s a brilliantly exciting time where we’re beginning to harvest the fruits of decades’ worth of breakthrough research undertaken by the world’s brightest scientists, and we’re seeing those advances in basic science translated to the clinic, where they’re making a difference in patients’ lives. Mark Garner, PhD.,

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Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia thalassemia in adult and paediatric patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Genotype Genetics 246

Drug Discovery World

SEPTEMBER 28, 2022

AI-based diagnostic software and clinical trials technology proved to be two of the most attractive health investments, alongside mental health and wellness and medical imaging companies. . But has its application been as revolutionary as industry bods predicted? . The simple answer is no. The unavailability of good data .

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Pharma Companies Drugs In-Vivo Genetics 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.

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Life Science Gene Editing Development Clinical Trials 119

XTalks

AUGUST 17, 2020

Cue Biopharma is a Cambridge, Massachusetts-based clinical-stage biotechnology company that is focused on developing injectable immune biologics using its proprietary Immuno-STAT (Selective Targeting and Alteration of T cells) platform. Cell-Based Immunotherapy vs. Immune Biologics. cells, receptors) to treat disease.

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Protein Engineer Immune Response In-Vivo 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

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Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

SEPTEMBER 6, 2022

And whilst clinical trials activity decreased by around 15% compared to 2020, last year still saw a number of regenerative medicines reach the bedside. Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7 billion, compared to $19.9 billion, compared to $19.9 million price point per dose.

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

MARCH 20, 2024

Around 30% of drugs show adverse reactions during human clinical trials despite promising pre-clinical studies. Their adaptability for genetic modifications further positions them as an optimal tool for exploring the mechanisms of action of toxic compounds.

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Research Clinical Trials Clinical Trials In-Vivo 59

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

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

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Gene Splicing Gene Recombinant DNA Technology DNA 130

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

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.

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