Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.

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Gene Therapy Drugs Gene Gene Editing 264

Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

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Gene Editing Genomics Genome Gene Therapy 101

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. Both therapies will be available in early 2024.

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Gene Therapy Gene FDA Approval In-Vivo 98

Pharmaceutical Technology

AUGUST 24, 2022

A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy. The ARCUS nuclease vector (GTP-506A) targets gene editing in the PCSK9 gene locus that is well characterised, while the therapeutic donor vector (GTP-506D) introduces the OTC gene to provide the required genetic correction.

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Gene Editing Gene Therapy Gene Genetics 130

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

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 59

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

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Gene Editing Gene Gene Therapy Scientist 105

pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs. The post 2nd Annual Gene Therapy Patient Engagement Summit appeared first on.

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Gene Therapy Gene Gene Editing Clinical Trials 52

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

Pharmaceutical Technology

APRIL 25, 2023

After missing the previous Q1 deadline, bluebird bio has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for its sickle cell disease gene therapy lovo-cel, based on an April 24 company announcement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Gene Editing Licensing 130

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. The Therapeutic Strategy Leads for Rare Disease at Worldwide Clinical Trials have been working alongside and championing these organizations for many years.

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Gene Gene Editing Gene Therapy Clinical Trials 130

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.

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Gene Editing Gene In-Vivo Genetics 52

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The current schedule is for trials to start in 2024, with first safety and efficacy data coming a year later.

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Gene Therapy Gene Gene Sequencing Gene Editing 52

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

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

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Pharmaceutical Technology

OCTOBER 13, 2022

Vita Therapeutics intends to use the funding to advance its lead pre-clinical programme VTA-100 for limb-girdle muscular dystrophy (LGMD2A) to clinical trials. VTA-100 has been designed to be an autologous therapy which combines gene correction as well as induced iPSC technology for repairing and replacing muscle cells for LGMD2A patients.

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Development Gene Editing Gene Gene Therapy 130

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Gene Therapy Gene Production Clinical Trials 52

The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced.

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Gene Therapy Gene Gene Editing Clinical Trials 52

pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. Aligning with the focus of the Cancer Moonshot Research Initiative , PACT aimed to “retrospectively analyse patient data” from past clinical trials with the goal of “predicting future patient outcomes.”.

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Research Gene Editing Development Gene 132

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.

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In-Vivo Gene Editing Trials Gene 52

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

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Research Gene Editing Gene Genetics 118

XTalks

NOVEMBER 30, 2023

The safety and efficacy of Cabenuva were established through two randomized, open-label, controlled clinical trials involving 1,182 HIV-infected adults who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) before initiating Cabenuva treatment. In November 2023, Hookipa Pharma Inc.,

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FDA Approval Gene Therapy Clinical Trials Gene 111

pharmaphorum

AUGUST 8, 2022

CRISPR Therapeutics and Vertex are also in the running with their gene-editing candidate CTX001, in phase 1/2 trials which are due to generate final results later this year. GBT’s inclacumab – another P-selectin antibody that could encroach on Adakveo – is in a pair of phase 3 trials due to generate results next year.

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pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. The post Bayer trumpets $1bn CRISPR deal with Mammoth Bio appeared first on.

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In-Vivo Gene Editing Gene Therapy Gene 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Manufacturing Gene Therapy Trials Gene 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.

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Gene Editing Gene In-Vivo Genetics 52

BioPharma Reporter

FEBRUARY 23, 2023

After voluntarily pausing a phase 1/2 trial due to safety concerns in January 2023, the gene edited cell therapy firm Graphite Bio has discontinued the development of nulabeglogene autogedtemcel (nula-cel), its lead candidate cell therapy for sickle cell disease, and has halved its workforce.

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Gene Editing Gene Trials Development 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. These companies are at various stages of research and clinical trials.

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Life Science Gene Editing Development Clinical Trials 119

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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Gene Therapy Gene Gene Editing Medicine 42

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. Among 20 recent trials, 17 experienced delays in meeting key milestones.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

WCG Clinical

OCTOBER 21, 2024

Clinical trials are the cornerstone of advancing medical research, but behind the science, the experiences of participants and their families offer invaluable insight into how research impacts real lives. Advances in research, such as gene editing and cellular therapies, have opened new treatment possibilities for both conditions.

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Clinical Trials Clinical Trials Trials Clinical Research 52

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.

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Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

OCTOBER 18, 2021

News that the Tofersen trial failed to meet its primary endpoint comes as a disappointment, but positive hints from secondary measures that earlier and longer administration may provide some positive clinical benefit. (1/2) The post Blow to Biogen as ALS hope tofersen flunks phase 3 trial appeared first on. 1/2) [link].

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Trials Gene Editing Gene Gene Therapy 45

pharmaphorum

FEBRUARY 17, 2022

Any near-term impact in Europe will be limited after Bluebird Bio pulled its Zynteglo (betibeglogene autotemcel) gene therapy for another rare blood disorder, beta thalassaemia, off the market in Germany after failing to secure agreement on pricing. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.

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Drugs Gene Therapy Gene Editing Gene 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.

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Genomics Genome Gene In-Vivo 40

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

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Life Science Gene Therapy Gene Manufacturing 52

Delveinsight

FEBRUARY 25, 2021

For most CNS disorders, the development of effective drug candidates is challenging as clinical trials are often long-lasting and with considerable expense. Positioning of Gene and Cell Therapies in Neurosciences. Gene and cell therapies hold the potential to provide a one-off cure. Race to be the first mover.

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Gene Gene Therapy Gene Editing Development 52