XTalks

AUGUST 30, 2022

The hemophilia A gene therapy Roctavian delivers a functional gene to enable the body to make an essential blood clotting protein on its own. A lack of sufficient functioning factor VIII and factor IX blood clotting proteins would result in the development of hemophilia A and hemophilia B, respectively.

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98

pharmaphorum

JUNE 17, 2021

Biogen has four other Alzheimer’s candidates in its clinical pipeline behind recently-approved Aduhelm, but one of them – anti-tau antibody gosuranemab – has just been canned. In Alzheimer’s, the protein runs amok, forming tangles that have been linked to cell damage and neuronal death.

Antibody 98

Antibody Protein Licensing Licensing 98

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

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Gene Gene Therapy Genetics DNA 262

Roots Analysis

NOVEMBER 30, 2021

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.

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Bioassay Gene Therapy Gene Development 52

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

Gene Therapy 52

Gene Therapy Gene Manufacturing Contract Manufacturing 52

XTalks

DECEMBER 8, 2020

has announced that it has acquired the rights to an investigational gene therapy, HMR59, from Hemera Biosciences, LLC designed to help treat a severe form of age-related macular degeneration (AMD). The gene therapy was developed to treat both dry and wet macular degeneration. Janssen Pharmaceuticals Inc.

Gene Therapy 52

Gene Therapy Gene Protein Development 52

pharmaphorum

JANUARY 13, 2022

Sanofi has joined a growing list of drugmakers going after alpha-synuclein targeting drugs for Parkinson’s disease, licensing a bispecific antibody from South Korea’s ABL Bio in a deal that could be worth more than $1 billion. An AbbVie antibody called ABBV-0805 successfully cleared a phase 1 trial but seems to have been shelved.

Antibody 98

Antibody Drugs Protein Licensing 98

pharmaphorum

DECEMBER 2, 2021

The drug – called UCB0599 – targets a protein called alpha synuclein that tends to get misfolded and accumulates into clumps in the brains of Parkinson’s patients, and is thought to damage neurons – somewhat analogous to amyloid and tau proteins in Alzheimer’s disease.

Antibody 111

Antibody Licensing Licensing Gene Therapy 111

XTalks

APRIL 1, 2025

In hemophilia patients, inhibitors are antibodies that interfere with the effectiveness of clotting factor replacement therapies. Qfitlia differs from traditional factor replacement therapies. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

AUGUST 23, 2022

It comprises messenger ribonucleic acid (mRNA) that encodes for the initial spike protein of SARS-CoV-2 along with mRNA that encodes for Omicron BA.4/BA.5 5 variant spike protein. According to pre-clinical findings, a booster dose of this vaccine elicited a robust neutralising antibody response against the variants Omicron BA.1,

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Vaccination Vaccine Gene Therapy Protein 130

Delveinsight

DECEMBER 3, 2020

Roche receives FDA authorization for the quantitative COVID-19 antibody test. Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

Pharmaceutical Technology

FEBRUARY 1, 2023

Since the FDA approved AstraZeneca’s monoclonal antibody therapy Synagis (palivizumab) 25 years ago, drug development successes for RSV have been few and far between. The two types of candidates currently in the pipeline can be categorised as monoclonal antibodies and vaccines.

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Vaccination Vaccine Antibody Development 162

pharmaphorum

FEBRUARY 3, 2022

Al Sandrock’s shock departure from Biogen in November sparked speculation about where he would show up next, and the answer came in today – gene therapy specialist Voyager Therapeutics. The setbacks effectively sent Voyager back to being a preclinical-stage company however, setting it back years from bringing therapies to market.

Gene Therapy 97

Gene Therapy Gene Clinical Trials Clinical Trials 97

pharmaphorum

APRIL 21, 2022

The pharma company has decided to end its collaboration with Swedish biotech BioArctic on alpha-synuclein-targeting antibody ABBV-0805, which was in early-stage clinical testing, as well as a portfolio of follow-up antibodies. AZ/Takeda are meanwhile running phase 2 trials of their MEDI 1342 antibody candidate.

Antibody 97

Antibody Protein Gene Therapy Pharma Companies 97

Pharmaceutical Technology

NOVEMBER 7, 2022

5 Omicron variants’ spike protein and 25µg encoding for the virus’ initial strain. Additionally, 28 days following inoculation, this vaccine showed enhanced neutralising antibody responses against Omicron BA.4/BA.5 Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 1 subvariant.

Vaccination 130

Vaccination Vaccine Gene Therapy Clinical Trials 130

Worldwide Clinical Trials

MAY 30, 2024

Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

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Gene Clinical Trials Clinical Trials Gene Therapy 204

Pharmaceutical Technology

MARCH 27, 2023

National Resilience will use the funding to establish a significant domestic biomanufacturing capacity and capabilities for vaccines, nucleic acids including mRNA, and biologics including antibodies, proteins and multi-specifics. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Manufacturing Vaccination Vaccine 130

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

Delveinsight

DECEMBER 22, 2020

FDA Puts uniQure’s Hemophilia B Gene Therapy on Leash. In a recent, uniQure faced backlash after its hemophilia B gene therapy got hit with clinical hold by the USFDA. . The Hemophilia B market has several therapies, however, no therapy has been able to kill the disease from its root.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Delveinsight

DECEMBER 10, 2020

Warding off Type 1 diabetes by aiming a T-cell protein. Scientists at the University of Utah School of Medicine have found a way to control that autoimmune response by aiming a protein that is vital for T-cell activation. The protein is known as OCA-B. UniQure/CSL haemophilia B gene therapy curbs bleeding in phase 3.

Gene Therapy 52

Gene Therapy Gene Vaccination Vaccine 52

XTalks

MAY 9, 2024

Genmab, a trailblazer in the field of antibody therapeutics, celebrates this milestone by reflecting on its impressive journey from a small-scale startup to a global leader in cancer treatment and beyond. Since then, innovative science has been at Genmab’s core, as we harness the power of human antibodies to improve the lives of patients.

Antibody 52

Antibody Clinical Trials Clinical Trials Medicine 52

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. It is an IgM antibody targeting Death Receptor 5 (DR5), and IGM is hoping to test Birinapant with IGM-8444 for the treatment of solid cancers later this year.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

XTalks

APRIL 12, 2021

Like other neurodegenerative disorders, such as Alzheimer’s disease and dementias, Parkinson’s disease research has been immensely challenging for biotechs, with a high rate of late-stage attrition in trials leading to a lack of disease-modifying therapies being approved. Axovant Gene Therapies. Prevail Therapeutics.

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Gene Therapy Gene Trials Development 96

The Pharma Data

FEBRUARY 1, 2021

The FDA issued straight-to-final guidance that provides sponsors of monoclonal antibody and other therapeutic protein COVID-19 treatments with recommendations for potency assays to ensure consistent product quality. The guidance does not cover vaccines, hyperimmune globulins, gene therapies, cell therapies or convalescent plasma.

Protein 40

Protein Gene Therapy Licensing Licensing 40

Pharmaceutical Technology

AUGUST 26, 2008

. “The inherent advantages of biopharma are that it allows you to pursue therapies against targets that are not small molecule therapies, for example by targeting protein-protein interactions and targeting GPCRs (G-protein coupled receptors),” he says. “It also had good maturing capability. .”

Antibody 100

Antibody Licensing Licensing Production 100

Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Beyond our expertise in small molecules, we have built a solid reputation with biopharmaceutical companies for the characterisation and analysis of monoclonal antibodies, therapeutic proteins and biosimilars.

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Development Production Manufacturing Vaccination 130

Delveinsight

AUGUST 6, 2020

ZyCoV-D is designed to introduce DNA that encodes for a viral protein into human cells. By ordering human cells to make the protein, ZyCoV-D could cause an immune response against the coronavirus. Taysha raises USD 95 Million to take 4 CNS gene therapies into the clinic.

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Allergies Vaccine Vaccination Gene Therapy 53

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

pharmaphorum

MARCH 9, 2022

Sanofi and Sobi’s haemophilia partnership has been under competitive pressure from new therapies like Roche’s antibody Hemlibra, so the two companies are hoping a new long-acting drug candidate can revitalise the franchise. The half life of the new drug – one of the main draws for Sanofi’s $11.6

Protein 98

Protein Gene Therapy Sales Regulation 98

Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. Some notable examples include: Monoclonal antibodies targeting cancer cells or tumor microenvironments, such as trastuzumab for breast cancer and pembrolizumab for melanoma.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

pharmaphorum

AUGUST 17, 2020

BMS is best known as an early pioneer of checkpoint inhibitor therapies, which work by flipping a biochemical switch on tumour cells that suddenly makes them visible to the immune system, allowing T-cells to mount an attack. The companies hope that this will cause the area around the tumour to become inflammatory and begin to work against it.

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Gene Therapy Clinical Trials Clinical Trials Gene 59

XTalks

NOVEMBER 30, 2023

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. This candidate, derived from a person’s own genetically modified cells, aims to restore the Gag-specific CD4+ T cell response in those receiving antiretroviral therapy.

FDA Approval 111

FDA Approval Gene Therapy Gene Clinical Trials 111

pharmaphorum

MARCH 31, 2021

The diagnostic imaging agents all target fibroblast activation protein (FAP), a molecule that is only expressed at low levels on healthy cells but is found much more abundantly in solid tumours. billion to buy targeted radioligand therapy Lu-PSMA-617 from Endocyte , shortly after encouraging results in a phase 2 prostate cancer trial. .

Licensing 98

Licensing Licensing Gene Therapy Sales 98

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. One strategy proposed the use of synthetic mRNA or modified mRNA analogs, which can enhance the stability and protein expression of mRNA.

Vaccination 52

Vaccination Vaccine Protein Antibody 52

Pharmaceutical Technology

JULY 29, 2022

The booster dose elicited a significantly higher neutralising antibody response against Omicron subvariants BA.4 5 spike protein components to their current vaccine compositions to create a two-component booster, so that the modified vaccines can potentially be used from later this year.

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Manufacturing Vaccination Vaccine Packaging 130

XTalks

OCTOBER 22, 2020

In this sense, pharmaceutical companies have developed different strategies to produce a wide variety of advanced therapy medicinal products (ATMP) and biological products, such as monoclonal antibodies, recombinant proteins, gene therapy viral vectors, CAR-T cells and oncolytic viruses, in order to deliver new medical needs in our society.

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Manufacturing Development Production Gene Therapy 98

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Hemlibra is a bispecific factor IXa- and factor X-directed antibody.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52