XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

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pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

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pharmaphorum

JUNE 27, 2022

The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to launch. The post BioMarin gene therapy for haemophilia on course for EU approval appeared first on.

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pharmaphorum

OCTOBER 6, 2021

Voyager Therapeutics has just landed a new gene therapy alliance with Pfizer that could go some way towards easing the pain of losing three other partners in the space of a couple of years. The post Pfizer agrees $630m deal to harness Voyager’s gene therapy tech appeared first on.

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pharmaphorum

JULY 20, 2022

Shares in VBL Therapeutics have lost around 78% of their value in pre-market trading after the company reported a phase 3 trial of its lead gene therapy VB-111 for ovarian cancer failed a pivotal trial. The post VBL craters as ovarian cancer gene therapy fails phase 3 test appeared first on.

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pharmaphorum

SEPTEMBER 13, 2021

AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a gene therapy for wet age-related macular degeneration (AMD) and other eye diseases. The post AbbVie spies potential in Regenxbio eye disease gene therapy appeared first on.

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BioPharma Reporter

MAY 9, 2023

Hansa Biopharma and Genethon have entered a research and development collaboration to test imlifidase as a gene therapy treatment in patients with pre-existing neutralizing antibodies.

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Gene Therapy Gene Antibody Development 94

Pharmaceutical Technology

JANUARY 17, 2023

The STAb therapy is based on a special type of bispecific antibody secretion. This antibody can detect targets on the tumour cell and the T cell. An artificial bridge created by these antibodies gets therapeutic T cells and tumour cells into contact. By Cytiva Thematic.

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FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. To be eligible for the therapy, patients must test negative for AAV5-targeted antibodies. Roctavian, a $2.9 SVB has forecasted around $2.2

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Pharmaceutical Technology

APRIL 14, 2023

Twist Bioscience CEO and co-founder Emily Leproust said: “With the dramatic increase in interest around cell and gene therapies, the addition of Twist CAR and TCR Libraries offers an important and differentiated tool to enable the development of the next generation of treatments for those suffering from a wide range of diseases.

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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.

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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. Last week, the first gene therapy for severe hemophilia A , Roctavian (valoctocogene roxaparvovec), developed by BioMarin Pharmaceutical Inc.,

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). million price tag of Elevidys, a one-time gene therapy. Options for managing the symptoms of DMD have been limited.

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Pharmaceutical Technology

APRIL 19, 2023

“Evidence is now available that most of the US population five years of age and older have antibodies to SARS-CoV-2, the virus that causes Covid-19, either from vaccination or infection, that can serve as a foundation for the protection provided by the bivalent vaccines.”

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FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

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Pharmaceutical Technology

JULY 15, 2022

According to the positive interim findings from the Phase II/III KidCOVE clinical trial, a two-dose primary series of mRNA-1273 offered a strong neutralising antibody response in the young children with a favourable safety profile. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

DECEMBER 18, 2022

According to the data from Phase II/III trials reported in November this year, the company’s bivalent Omicron-targeting booster candidates, mRNA-1273.214 and mRNA-1273.222, showed to induce an antibody response superior to a booster dose of a prototype vaccine against Omicron (BA.4-BA.5), 5), mRNA-1273. By Cytiva Thematic.

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Pharmaceutical Technology

NOVEMBER 14, 2022

In a field dominated by antibodies and small molecules, two cell-therapy based approaches have come under the spotlight for showing early signs of efficacy in treating lupus. Apart from the monoclonal antibodies and small molecules, researchers have long held an interest in exploring cell therapies for autoimmune conditions.

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Pharmaceutical Technology

JUNE 29, 2022

These subjects received a minimum of two lines of systemic therapy previously, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. FL is a kind of indolent non-Hodgkin lymphoma (iNHL).

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Pharmaceutical Technology

JULY 18, 2022

According to positive interim data from the Phase II/III KidCOVE clinical trial, the vaccine offered a strong neutralising antibody response in children of the age group of six months to five years following administration of the two-dose initial regimen of the mRNA-1273 vaccine.

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Pharmaceutical Technology

AUGUST 15, 2022

In baseline seronegative subjects, the updated vaccine offered a superior neutralising antibody response against Omicron (BA.1) Irrespective of previous infection or age, the vaccine-induced potent neutralising antibody responses against the BA.4 Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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pharmaphorum

APRIL 16, 2021

The 2 nd Gene Therapy Immunogenicity Summit is the industry’s definitive forum enabling you to better modulate, measure and predict immune response to your gene therapy candidate. Performances of biosafety analytical assays commonly used in gene therapy development.

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pharmaphorum

NOVEMBER 30, 2020

Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies. Neutralising antibodies (NAbs) present a unique challenge to researchers looking to treat patients with AAV treatment.

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Gene Therapy Gene Antibody Clinical Trials 105

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. The post Biogen abandons tau antibody for Alzheimer’s after phase 2 miss appeared first on.

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Pharmaceutical Technology

NOVEMBER 22, 2022

Despite China producing a significant proportion of the world’s API supply (mostly small molecule), it manufactures relatively few biosimilar and innovator drugs and no cell and gene therapies for the western markets of Europe and the US despite investments and an increasing number of startups to improve innovative manufacture.

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BioPharma Reporter

DECEMBER 2, 2021

Several contract development and manufacturing organization (CDMO) supported biopharma projects have been flagged in the past month. We take a closer look.

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BioTech 365

FEBRUARY 10, 2021

Launch of Novel Gene Therapy Company, VectorY, to Develop Innovative Vectorized Antibodies Launch of Novel Gene Therapy Company, VectorY, to Develop Innovative Vectorized Antibodies Fully integrated gene therapy offering, including platform technology, pipeline development, and manufacturing capabilities Targeting muscular and … Continue reading (..)

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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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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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pharmaphorum

MARCH 8, 2022

Voyager Therapeutics has signed another big pharma partner for its TRACER adeno-associated virus (AAV) vector technology, designed to improve the safety of gene therapies, with Novartis taking an option on three neurological disease programmes. billion including fees and milestones.

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Gene Therapy Gene Antibody 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.

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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. The company has developed various assets based on lentivirus vectors.

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BioTech 365

DECEMBER 1, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Wilson, Penn ink Regeneron pact to use gene therapy tech to deliver COVID-19 antibodies.Wilson, Penn ink Regeneron pact to use gene therapy tech to deliver COVID-19 antibodies … Continue reading →

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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.

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Gene Therapy Gene Protein Development 52

STAT News

DECEMBER 22, 2022

Merck has agreed to pay $175 million in cash to Kelun-Biotech, a division of the Chinese pharmaceutical firm Sichuan Kelun Pharmaceutical, for the rights to seven preclinical antibody-drug conjugates, a type of cancer drug that has seen some big successes recently. billion.

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