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

Pharma Mirror

FEBRUARY 18, 2021

Maculus plans to raise USD3 million on a USD8 million valuation for a 12-18 month period, including initial in-vivo animal studies and completion of in-vitro / in-vivo drug elution studies. Maculus achieved the breakthrough using a patented novel tunable biodegradable proprietary product, MacuBloc.

Drug Delivery 130

Drug Delivery In-Vivo In-Vitro Drugs 130

Drug Patent Watch

JANUARY 14, 2025

The Generic Drug Revolution: Setting the Stage Before we dive into the nitty-gritty, let’s take a moment to appreciate the impact of generic drugs. Did you know that generic drugs now make up about 90% of all prescription drug purchases in the United States, while accounting for only 20% of prescription drug spending[1]?

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Generic Drugs Development Drugs Manufacturing 87

Pharmaceutical Technology

FEBRUARY 23, 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 Artificial Intelligence in Pharmaceuticals: In-silico drug discovery. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189

Drugs DNA In-Vivo In-Vitro 189

XTalks

NOVEMBER 25, 2024

Shorla Oncology has received US Food and Drug Administration (FDA) approval for Imkeldi, an oral solution of imatinib designed to treat specific forms of leukemia and other cancers. The drug also reduces exposure risks for healthcare workers by improving handling safety in oncology clinics.

In-Vivo 105

In-Vivo FDA Approval In-Vitro Drugs 105

BioSpace

OCTOBER 21, 2021

The experimental drug, NTLA-2001, is being developed for the treatment of transthyretin (ATTR) amyloidosis.

In-Vivo 89

In-Vivo Drugs Development 89

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.

Gene Editing 111

Gene Editing In-Vivo Drugs Clinical Trials 111

Pharmaceutical Technology

JANUARY 4, 2023

The China National Medical Products Administration’s (NMPA) Center for Drug Evaluation (CDE) has granted clearance for Transcenta’s investigational new drug (IND) application for its TST003 to treat solid tumours. TST003 is a first-in-class Gremlin1 targeting humanised monoclonal antibody.

Antibody 242

Antibody In-Vivo In-Vitro Protein 242

Bio Pharma Dive

DECEMBER 18, 2024

The Bill & Melinda Gates Foundation will invest up to $50 million in Tessera’s in vivo sickle cell therapy. Elsewhere, Cara agreed to a reverse merger and J&J had a drug application rejected.

In-Vivo 130

In-Vivo Drugs 130

Pharmaceutical Technology

SEPTEMBER 19, 2022

The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio ’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. The treatment is indicated for usage in boys aged four to 17 years with CALD. thalassemia.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

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.

Gene Therapy 130

Gene Therapy Gene Drugs In-Vivo 130

Pharmaceutical Technology

APRIL 4, 2023

Vertex Pharmaceuticals and CRISPR Therapeutics have completed the rolling submission of biologics licence applications (BLAs) to the US Food and Drug Administration (FDA) for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

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Gene Therapy Gene Editing In-Vivo Gene 246

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.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

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

Pharmaceutical Technology

MAY 16, 2023

Nuvectis Pharma has secured approval from the US Food and Drug Administration (FDA) for its investigational new drug application (IND) for NXP900. Nuvectis now has two precision oncology, clinical-stage drug candidates, each with several potential opportunities in cancers that represent serious conditions of unmet medical need. “We

In-Vivo 130

In-Vivo In-Vitro Clinical Trials Clinical Trials 130

Pharmaceutical Technology

AUGUST 21, 2022

The China National Medical Products Administration (NMPA) has granted clearance for Ascletis Pharma’s Investigational New Drug (IND) application of its drug candidate, ASC10, for Covid-19. These two drugs, following oral dosing, are quickly and fully transformed into the same active metabolite ASC10-A or ? -D-N4-hydroxycytidine

In-Vivo 162

In-Vivo RNA Clinical Development Drugs 162

Camargo

NOVEMBER 11, 2020

When the FDA requires a product’s labeling to include a boxed warning (also called a “black box warning” because the text is surrounded by black border), the potential market value of the drug often drops severely. Presumably, Antares’ long-established 90-person urology sales force can achieve pull-thru of this drug product.

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Development Bioequivalency Generic Drugs Production 242

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.

Gene Editing 97

Gene Editing In-Vivo Gene Sequencing Gene 97

pharmaphorum

NOVEMBER 18, 2022

The BRILLIANCE study of EDIT-101 in Leber congenital amaurosis type 10 (LCA10) – an inherited form of blindness – was reported to be the first ‘in vivo’ CRISPR/Cas9 medicine to be administered to a patient when it started in 2020. The post After disappointing trial, Editas puts lead CRISPR drug up for sale appeared first on.

Sales 92

Sales In-Vivo Trials Gene Editing 92

World of DTC Marketing

JANUARY 31, 2021

Porter points out that R&D seems to have been replaced with mergers and acquisitions, but she misses the big picture by focusing solely on drug costs. So where to start… Congresswoman Porter may want to look at the percentage of generic drugs available virus branded (89%). In pharma R&D returns have declined to 1.8

In-Vivo 187

In-Vivo Generic Drugs Drugs Gene Therapy 187

pharmaphorum

FEBRUARY 13, 2022

German drugmaker Merck KGaA has reached an agreement to pilot an artificial intelligence platform developed by Quris that aims to identify potential safety problems with new therapeutic candidates as early as possible in the drug discovery process.

Drugs 98

Drugs In-Vivo In-Vitro Pharma Companies 98

XTalks

APRIL 14, 2022

Conventional in vitro and in vivo studies are used in the drug development pipeline. This involves the use of an individualized computer simulation for medical device or drug development. Therefore, companies can evaluate their drug candidates or medical device products in “virtual patients” before assessing them in humans.

Trials 98

Trials In-Vivo In-Vitro Clinical Trials 98

Pharmaceutical Technology

MAY 24, 2023

This promise and growth are expected to continue as CGT, which have the potential to cover 70% of oncology drugs, are being developed to treat further disease types such as solid tumors. CGT drugs fall into two major categories: autologous and allogeneic. CGT trials operate with much lower volumes of manufactured final product.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Pharmaceutical Technology

MAY 5, 2023

TFF Pharmaceuticals and NIEHS will investigate the TFF-HMW-HA formulations’ therapeutic efficacy and pharmacokinetics using in vitro and in vivo models of respiratory diseases.

Development 162

Development In-Vivo In-Vitro Engineer 162

pharmaphorum

AUGUST 10, 2020

Gilead has filed its COVID-19 drug remdesivir with the FDA, to treat patients with severe disease, under the brand name Veklury. The drug is currently available to US patients under an Emergency Use Authorisation for treatment of hospitalised patients with severe COVID-19. Feature image courtesy of Rocky Mountain Laboratories/NIH.

Drugs 99

Drugs In-Vivo In-Vitro Contract Manufacturing 99

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

Pharmaceutical Technology

NOVEMBER 4, 2022

The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. at closing.

In-Vivo 130

In-Vivo In-Vitro Genetics Drugs 130

pharmaphorum

MARCH 18, 2025

AstraZeneca plans to acquire Belgian in vivo cell therapy biotech EsoBiotec and has signed a pair of licensing deals with drug delivery firm Alteogen.

In-Vivo 66

In-Vivo Drug Delivery Licensing Licensing 66

Pharmaceutical Technology

SEPTEMBER 14, 2022

The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.

In-Vivo 130

In-Vivo Clinical Trials Clinical Trials Engineer 130

pharmaphorum

JANUARY 29, 2021

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. Zinc fingers. billion in funding.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Scienmag

JANUARY 10, 2022

An international research group at the RIKEN Cluster for Pioneering Research (CPR) has successfully treated cancer in mice using metal catalysts that assemble anticancer drugs together inside the body.

In-Vivo 63

In-Vivo Drugs Research 63

Pharmaceutical Technology

FEBRUARY 13, 2023

However, there is a high level of attrition during the pharmaceutical research and development process, which is an indicator of the vast number of potential drug substances considered for progression. With all this in mind, how do drug developers know which strategy and approach is right for their molecule at this early stage?

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Development In-Vivo Drugs Clinical Trials 130

BioTech 365

SEPTEMBER 15, 2020

In vivo efficacy on a par with gold standard deruxtecan-based ADCs Poster to be presented at World ADC meeting on 17 September 2020 AMSTERDAM–(BUSINESS WIRE)–Synaffix B.V., a biotechnology company enabling antibody-drug conjugates (ADCs) with best-in-class therapeutic index, based on proprietary … Continue reading →

In-Vivo 40

In-Vivo Antibody Drugs 40

Pharmaceutical Technology

FEBRUARY 1, 2023

The US Food and Drug Administration (FDA) has granted approval for Ellipses Pharma’s investigational new drug (IND) application for EP0042 to treat acute myeloid leukaemia (AML). The dual FLT-3 and Aurora kinase inhibitor EP0042 is being developed to treat AML patients who have developed FLT3 inhibitor resistance.

FDA Approval 130

FDA Approval In-Vivo In-Vitro Trials 130

Pharmaceutical Technology

MAY 10, 2023

The research teams will examine the use of CytoMed’s allogeneic gdTc on multiple AML and breast cancer subtypes in vitro and in vivo. Data from the study are expected to support an investigational new drug application with the US Food and Drug Administration for the allogeneic use of gdTc to treat blood and solid cancers.

Gene Therapy 147

Gene Therapy In-Vivo In-Vitro Gene 147

Worldwide Clinical Trials

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. In June 2023, the U.S. In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

Camargo

DECEMBER 22, 2021

Informed by today’s competitive drug development landscape, as well as by the sponsor’s strategic goals and capabilities, a portfolio analysis involves assessing product concept and differentiation, strategic planning, factoring in unmet patient needs, and estimating timelines and expenses. Incentive Programs. Clinical Pharmacology.

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Bioequivalency Production Genotoxicity In-Vivo 169