pharmaphorum

JANUARY 6, 2022

Roivant is the latest pharma group to take a position in the emerging field of therapies targeted at RNA splicing, licensing exclusive rights to a drug developed by a unit of Japanese drugmaker Eisai. . The post Roivant joins RNA splicing push with Eisai deal appeared first on.

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XTalks

JANUARY 3, 2024

Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to develop gene therapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). The total value of the deal could potentially reach $1.2 Sandrock, Jr., MD, PhD, CEO of Voyager.

Gene Therapy 52

Gene Therapy Gene Licensing Licensing 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

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pharmaphorum

MAY 11, 2022

The pharma group will provide funding for the research in return for exclusive license options on the programmes. billion takeover of gene-silencing specialist Dicerna Pharma last year, another deal aimed at extending its R&D capabilities by bolting on a new platform technology – in this case RNA interference (RNAi) drugs.

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pharmaphorum

JANUARY 5, 2023

Nonetheless, Moderna has previously been involved in licensing deals to advance its technology. The data was from their phase 2b trial of a personalised vaccine consisting of 34 mRNAs, each targeting mutations – identified by gene sequencing – thought to be driving a patient’s cancer.

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XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

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pharmaphorum

MAY 26, 2022

Gümrükcü is a specialist in gene expression profiling in stem cells, and has been working on ways to deliver stem cell transplants to treat cancer without the need for high-dose induction chemotherapy to destroy the patient’s bone marrow. ” If convicted, they face mandatory life in prison or the death penalty.

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Gene Expression RNA Containment Licensing 105

pharmaphorum

FEBRUARY 9, 2021

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells. Regulus meanwhile remains a microRNA player, but has suffered a number of setbacks.

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Trials Gene Silencing RNA Gene Expression 59

Delveinsight

AUGUST 5, 2021

The acquisition is the recent sign that large pharmaceutical companies view messenger RNA, which BioNTech and Moderna utilized to develop the COVID-19 vaccines now cleared for use in dozens of countries, as a crucial drug-making platform. Fibroblasts are part of the platform fueling cell-based gene therapy biotech Castle Creek.

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pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

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XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. million, compared to $1.1 million for the same period in 2022.

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pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Oxlumo (lumasiran) was also approved in the European Union last week, making it the third from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

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FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

NOVEMBER 2, 2022

CSL’s vaccines unit CSL Seqirus division is paying Arcturus $200 million upfront to gain access to its self-amplifying mRNA technology platform and exclusive licenses to several vaccine candidates, with another $4.3 billion on offer in milestones if the programmes meet development and commercial objectives.

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The Pharma Data

SEPTEMBER 30, 2021

Liu’s team was ready to encapsulate the agonist of the stimulator of interferon genes (STING) within liposomal particles to yield the adjuvant named NanoSTING. the corporate has an exclusive license agreement with UH with reference to the property covering intranasal vaccines and STING agonist technologies.

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Vaccine Vaccination Immune Response Licensing 52

The Pharma Data

MARCH 11, 2021

Sanofi Pasteur, the vaccines global business unit of Sanofi, and Translate Bio (NASDAQ: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company, today announced the start of the Phase 1/2 clinical trial for MRT5500, an mRNA vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. About the Phase 1/2 clinical trial.

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Clinical Trials Clinical Trials Vaccine Vaccination 52

The Pharma Data

NOVEMBER 29, 2020

Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

AUGUST 3, 2021

Accelerates development of current Sanofi licensed programs in vaccines and potential to explore other therapeutic areas Fast tracks establishment of Sanofi’s recently announced mRNA Center of Excellence Full integration upgrades drug formulation capabilities and enhances US talent in a promising new technology. About Translate Bio.

Vaccine 52

Vaccine Vaccination Development Clinical Trials 52

The Pharma Data

JUNE 26, 2021

Sanofi Pasteur, the vaccines global business unit of Sanofi and Translate Bio (NASDAQ: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company, have initiated a Phase 1 clinical trial evaluating an mRNA-based investigational vaccine against seasonal influenza. JUNE 22 , 2021.

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Clinical Trials Clinical Trials Vaccine Vaccination 52

The Pharma Data

JANUARY 12, 2021

Additionally, Jounce exclusively licensed worldwide rights to JTX-1811, a monoclonal antibody targeting CCR8 and designed to selectively deplete T regulatory cells in the tumor microenvironment, to Gilead Sciences, Inc.

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Clinical Trials Clinical Trials Antibody Trials 52

The Pharma Data

MARCH 4, 2021

Dravet syndrome is most commonly caused by a genetic mutation in the SCN1A gene and affects approximately 1 in 15,000 to 1 in 21,000 people in the United States. Dravet and Lennox-Gastaut syndrome typically become apparent during infancy or early childhood and are highly refractory to many antiseizure medications.

Development 52

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The Pharma Data

AUGUST 29, 2020

This analysis confirms that as a small interfering RNA (siRNA), inclisiran provides a remarkably consistent treatment profile. Inclisiran will potentially be the first and only LDL-C-lowering small-interfering RNA (siRNA) treatment. A century of cholesterol and coronaries: from plaques to genes to statins. 2015;161(1):161–172.

Medicine 52

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XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. Late in 2022, AbbVie partnered with HotSpot Therapeutics and announced an exclusive worldwide collaboration and option to license agreement for their interferon regulatory factor 5 (IRF5) inhibitor program for autoimmune disease treatment.

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Sales Manufacturing FDA Approval Life Science 98

XTalks

MAY 29, 2024

In its acquisitions, J&J also acquired antibody-drug conjugate (ADC) developer Ambryx and cardiac med tech Laminar along with more than 50 smaller, early-stage licensing deals and partnerships. Among partnerships and acquisitions, GSK in-licensed two ADCs targeting B7-H3 and B7-H4 from Hansoh Pharma in China. billion In 2023, F.

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Pharma Companies Sales FDA Approval Vaccination 52

The Pharma Data

APRIL 30, 2021

AbbVie announced the FDA accepted its New Drug Application (NDA) for atogepant, an investigational orally administered calcitonin gene-related peptide (CGRP) receptor antagonist (gepant), for the preventive treatment of migraine in adults who meet criteria for episodic migraine. and by AbbVie outside of the U.S.

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FDA Law Blog

OCTOBER 24, 2024

Rather, developers of diagnostic tests and, indeed of any product that relies on free access to gene sequence and other biomarker information, should pay also close attention, as PERA would overturn longstanding judicial precedent. By 2010, about 2000 isolated human genes had been patented in the U.S. Supporters of AMP v.

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XTalks

FEBRUARY 20, 2025

In hereditary angioedema (HAE), for example, donidalorsen an investigational RNA-targeted prophylactic treatment has demonstrated impressive efficacy. The FDA has accepted a Biologics License Application (BLA) and designated Priority Review to UX111, offering a potential option for affected families.

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