Pharmaceutical Technology

JULY 14, 2022

Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II (MPSII) or Hunter syndrome. The company noted that this gene therapy is the fourth one to receive orphan drug designation.

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Gene Therapy Gene Drugs In-Vivo 130

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

Pharmaceutical Technology

SEPTEMBER 19, 2022

The latest development is based on findings from the single-arm, open-label Phase II/III ALD-102 (Starbeam) and Phase III ALD-104 clinical trials. The treatment is indicated for usage in boys aged four to 17 years with CALD. 1) in Skysona-treated and untreated subjects. 1 versus 43% in untreated subjects. thalassemia.

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FDA Approval In-Vivo Gene Therapy Clinical Trials 246

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.

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Gene Therapy Gene Editing In-Vivo Gene 246

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 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.

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Gene Therapy Drugs Gene Gene Editing 264

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

pharmaphorum

FEBRUARY 1, 2024

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, pointing to the potential of CRISPR-based drugs delivered in vivo to treat diseases.

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Gene Editing In-Vivo Drugs Clinical Trials 111

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins.

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Gene Splicing Gene DNA Recombinant DNA Technology 130

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

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95

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.

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Gene Editing In-Vivo Gene Sequencing Gene 97

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

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. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Gene Therapy Gene FDA Approval In-Vivo 98

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene In-Vivo Life Science 130

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. It will also use a variation of CRISPR/Cas9 known as CRISPR/Casx which is smaller, making it easier to deliver using viral vectors for example, and more efficient at modifying genes in the body.

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Gene Editing Gene In-Vivo Clinical Trials 69

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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In-Vivo Generic Drugs Drugs Gene Therapy 187

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

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.

Gene Editing 59

Gene Editing Gene Gene Therapy Drugs 59

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.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

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.

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Gene Editing In-Vivo DNA In-Vitro 105

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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In-Vivo Gene Editing 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.

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Life Science Gene Editing Development Clinical Trials 119

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

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. Our focus is now on accelerating progress to help realise the potential for durable and curative cell and gene therapies. 18, 2021 09:00 UTC. A total of $15.4

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Gene Therapy Gene In-Vivo Genetics 40

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 Gene 52

Roots Analysis

FEBRUARY 27, 2024

The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. The global genome editing market is anticipated to grow at a CAGR of 12.6%

Genomics 40

Genomics Genome Gene In-Vivo 40

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.

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Gene Gene Therapy Gene Expression Regulation 40

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. Avrobio gene therapy eradicates toxic substrate in Fabry patient. Enhancing the response to immuno-oncology in pancreatic cancer. They reported their observations in the journal JCI Insight.

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Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

JANUARY 27, 2021

a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. The first US subjects in the PROOF-HD trial were enrolled in October 2020.

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Clinical Trials Clinical Trials Trials In-Vivo 40

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. But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion.

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In-Vivo Marketing Engineer Genetics 52

The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

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Medicine Development Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 22, 2020

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years’ experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. MILAN, Italy and NEW YORK, Nov. About Genenta Science.

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Gene Therapy Gene In-Vivo Clinical Trials 40

XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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Gene Editing Manufacturing Gene In-Vivo 52

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. Recently clinical investigators have found a way to turn what was an undesirable side effect in cancer patients into a clinical benefit for persons with certain autoimmune conditions.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system.

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Marketing Gene Drugs Protein 52

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 40