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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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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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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Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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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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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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Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

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Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The patient’s modified blood stem cells, now including a working copy of the SGSHgene, are then given back to the patient.

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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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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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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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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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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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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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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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Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% 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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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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Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

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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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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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Drug Discovery World

APRIL 15, 2024

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

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XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

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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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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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Drug Discovery World

MAY 6, 2024

billion on R&D during 2021, with Germany and Switzerland being the biggest contributors, and around one fourth of clinical trials worldwide were performed in Europe 1. The city is host to many innovative biotech companies, including relative newcomers Orexa, a company that recently launched its first Phase II clinical trial.

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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. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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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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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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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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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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Drug Discovery World

DECEMBER 20, 2022

There will be greater emphasis on diversity in clinical trials. A focus on the representation of diverse patient populations in clinical trials will be a very good thing for the industry, but could be challenging. DDW’s Diana Spencer summarises the key trends likely to impact the market in 2023. .

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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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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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Drug Discovery World

SEPTEMBER 6, 2022

The cell and gene therapy industry has experienced exponential growth in the last three years and as funding for the field continues to break records there are no signs of a slowdown. With 2,261 ongoing clinical trials in regenerative medicine, the FDA expects to approve 1 between 10 and 20 new cell and gene therapies a year by 2025.

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