pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. For now, the list price hasn’t been set and will depend on discussions with reimbursement authorities, according to the biotech.

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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

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. Voyager meanwhile has had its own difficulties, albeit not related to safety.

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Gene Therapy Gene Antibody Manufacturing 105

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

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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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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Gene Therapy Gene Immune Response Antibody 98

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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Gene Therapy Gene FDA Approval Production 119

Pharmaceutical Technology

APRIL 14, 2023

Researchers will now be able to speed up the creation of tailor-made libraries that can help in identifying and developing new cell therapies. Both libraries feature up to 10,000 gene fragment combinations, and allow the high-throughput screening and characterisation of new and known sequence variants to develop therapeutic solutions.

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Development Gene Therapy Gene Antibody 246

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. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Gene Therapy Gene FDA Approval Antibody 98

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

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 98

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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Gene Therapy Gene Clinical Trials Clinical Trials 98

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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Genome Genomics Medicine Gene Therapy 244

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). It is indicated for patients who do not have a pre-existing medical reason preventing treatment with the therapy.

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Gene Therapy Gene Protein FDA Approval 98

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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Development Gene Therapy Antibody Gene 276

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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Gene Therapy Gene Trials Clinical Trials 98

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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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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Gene Therapy Vaccination Vaccine Clinical Trials 304

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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Vaccination Vaccine Gene Therapy Antibody 264

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. The impact of innate and anti-transgene immunogenicity on AAV vector-mediated gene transfer with Genethon.

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Gene Therapy Gene Immune Response Antibody 52

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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Gene Therapy Clinical Trials Clinical Trials Genetics 278

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

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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Manufacturing Gene Therapy Gene Contract Manufacturing 162

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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Antibody Gene Therapy Manufacturing Trials 362

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.

Gene Therapy 105

Gene Therapy Gene Antibody Clinical Trials 105

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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Vaccination Vaccine Gene Therapy Antibody 246

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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Vaccination Vaccine Immune Response Clinical Trials 214

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

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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Gene Therapy Gene Manufacturing Contract Manufacturing 52

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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Gene Therapy Antibody Gene Manufacturing 93

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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Gene Therapy DNA Gene Genetics 246

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 (..)

Gene Therapy 40

Gene Therapy Antibody Gene Development 40

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

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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Antibody Protein Licensing Licensing 98

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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Gene Therapy Gene Antibody Research 40

Worldwide Clinical Trials

MAY 30, 2024

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications.

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