DNA, Gene Therapy, Genetics and In-Vivo - Clinical Research Informer

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

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.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Moderna partners with Life Edit for mRNA gene editing therapies

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’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

The challenges and trends of cell & gene therapies

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9

Gene Therapy

Gene Therapy Gene Manufacturing Medicine

Overcoming diverging regulatory expectations to bring CGTs to market

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy

Gene Therapy Marketing Gene DNA

Gene editing: beyond the hype

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.

Gene Editing

Gene Editing Gene Gene Therapy In-Vivo

Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

MAY 28, 2024

Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. An expected and well-known side effect of these B cell-targeted therapies is “B cell aplasia”— i.e. partial or complete depletion of B cells from circulation and immune organs.

In-Vivo

In-Vivo Clinical Trials Clinical Trials FDA Approval

This week in drug discovery (1-5 July)

Drug Discovery World

JULY 5, 2024

The last few days have seen some interesting developments related to gene editing, including the discovery of a new mechanism for genetic programming and evidence in favour of redosing CRISPR-based therapies, as well as significant investment and new indications for gene therapies.

Gene Therapy

Gene Therapy Gene Editing In-Vivo Gene

Biologics – The Next Step in Revolutionary Medication

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.

Contract Manufacturing

Contract Manufacturing Manufacturing Protein Bacteria

Data demonstrates superiority of circular RNA vs linear mRNA

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. DNA vectors compared to standard linear mRNA-based expression. “We

RNA

RNA In-Vivo Gene Therapy Genetic Disease

Gene Switch: A Novel Platform for Switching Genes On and Off

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

Gene Gene Therapy Gene Expression Regulation

Delivering on the promise of gene editing

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

Gene Editing Gene In-Vivo Genome

Life Science Trends to Look Out for in 2024

XTalks

DECEMBER 20, 2023

This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease. Below are some of the key trends that will be shaping the future of the life sciences in 2024 and beyond. The technologies enable the analysis of vast amounts of biological data at speeds and accuracies previously unimaginable.

Life Science

Life Science Gene Editing Development Clinical Trials

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

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

Clinical Trials Clinical Trials Gene Therapy Trials

Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

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

Gene Therapy Gene In-Vivo Licensing

CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. CRISPR also addresses another limitation in CAR T therapies related to the insufficient quantity and poor quality of autologous T cells.

DNA

DNA Genome Genomics RNA

What to expect from PEGS Europe 2023: Day 2

Drug Discovery World

NOVEMBER 9, 2023

Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’. Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting.

Engineer

Engineer Antibody Protein In-Vivo

Cryomacs Freezing Bags – A Critical Component in the Successful Delivery of Biological Samples and Therapies

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.

Engineer

Engineer Containment In-Vivo Production

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector.

Gene Editing

Gene Editing Gene Engineer DNA

Clinical Research Informer

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Moderna partners with Life Edit for mRNA gene editing therapies

Trending Sources

The challenges and trends of cell & gene therapies

Overcoming diverging regulatory expectations to bring CGTs to market

Gene editing: beyond the hype

Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

This week in drug discovery (1-5 July)

Biologics – The Next Step in Revolutionary Medication

Data demonstrates superiority of circular RNA vs linear mRNA

Gene Switch: A Novel Platform for Switching Genes On and Off

Delivering on the promise of gene editing

Life Science Trends to Look Out for in 2024

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

CRISPR breakthroughs: New solutions for common diseases

What to expect from PEGS Europe 2023: Day 2

Cryomacs Freezing Bags – A Critical Component in the Successful Delivery of Biological Samples and Therapies

The use of base editing in stem-cell based therapies

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