XTalks

MAY 1, 2023

This makes Qalsody the first approved treatment to target a genetic cause of ALS. Tofersen was discovered by Ionis Pharmaceuticals, and Biogen licensed tofersen from Ionis under a collaborative development and license agreement. The recommended dosage of Qalsody is 100 mg (15 mL) per administration. What is SOD1 -ALS?

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The Pharma Data

DECEMBER 14, 2020

15, 2020 (GLOBE NEWSWIRE) — Burning Rock Biotech Limited (NASDAQ: BNR, the “Company” or “Burning Rock”) today announced that it entered into an exclusive licensing agreement with Oncocyte Corporation (NYSE American: OCX) to bring DetermaRx , a risk stratification test for early stage lung cancer patients, to China.

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. One substantial obstacle is from the additional regulatory requirements of gene therapies containing genetically modified organisms (GMOs). Rare diseases can often be progressive, chronic and fatal.

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The Pharma Data

JANUARY 21, 2021

Session: Expanding Targetable Genetic Alterations in NSCLC Mini Oral Session Date and Time: January 31, 2021, 14:20 SGT (January 30, 2021 10:20 p.m. PT) Presentation Number: MA11.04. This pipeline has the potential to transform the company in the near future. SPECTRUM PHARMACEUTICALS, INC.

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The Pharma Data

MARCH 14, 2021

This is much lower than would be expected to occur naturally in a general population of this size and is similar across other licensed COVID-19 vaccines. The monthly safety report will be made public on the European Medicines Agency website in the following week, in line with exceptional transparency measures for COVID-19.

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Vaccination Vaccine Licensing Licensing 52

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Spinal Muscular Atrophy (SMA) ? Source link: [link] /.

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The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. This press release contains forward-looking statements, which are generally statements that are not historical facts.

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The Pharma Data

NOVEMBER 13, 2020

cVDPVs are rare and occur if the weakened strain of the poliovirus contained in the oral polio vaccine (OPV) circulates among under-immunized populations for a long time. If not enough children are immunized against polio, the weakened virus can pass between individuals and over time genetically revert to a form that can cause paralysis.

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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. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, Neuroscience, and Gastroenterology (GI). Important Notice From Takeda.

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The Pharma Data

MAY 27, 2022

It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. AstraZeneca COVID-19 vaccine was invented by the University of Oxford. About AstraZeneca.

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pharmaphorum

SEPTEMBER 18, 2020

Meanwhile, the search for the ‘right target’ led the company to collaborate with Cancer Research UK and launch the Functional Genomics Centre , a centre of excellence for genetic screening, cancer modelling and big data processing aimed at accelerating the discovery of new cancer medicines, including those based on CRISPR technology.

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Delveinsight

AUGUST 17, 2021

Food and Drug Administration, which could have entered the ultra-rare genetic disease market. Seagen will pay USD 200 million upfront to RemeGen and also secure an exclusive worldwide license agreement of upto USD 2.4 Paris-based Pharmaceutical Ipsen draws out the NDA for palovarotene from the U.S The rarity of disease – 1.36

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XTalks

AUGUST 15, 2023

Akeega is a dual-action tablet (DAT) that contains the company’s androgen receptor-targeting hormone therapy Zytiga (abiraterone acetate) and PARP inhibitor (poly [ADP-ribose] polymerase inhibitor) niraparib. Niraparib is also sold by GSK under the name Zejula in other indications. In April 2016, Janssen Biotech, Inc.

FDA Approval 52

FDA Approval Gene Hormones Drugs 52

The Pharma Data

MAY 13, 2021

Neither ClickBank nor the author are engaged in rendering medical or similar professional services or advice via this website or in the product, and the information provided is not intended to replace medical advice offered by a physician or other licensed healthcare provider. Individual results, including amount and time, will vary.

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The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. It may also allow for priority or rolling review of a company’s Biologics License Application (BLA). LEXINGTON, Mass., About LogicBio Therapeutics. Forward Looking Statements.

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The Pharma Data

NOVEMBER 17, 2021

It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. COVID-19 Vaccine AstraZeneca is known as Vaxzevria in Europe. About AstraZeneca.

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Vaccination Vaccine Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 2, 2020

This communication contains certain “forward-looking statements” within the meaning of the U.S. AD04 is also believed to have the potential to treat other addictive disorders such as opioid use disorder, gambling, and obesity. www.adialpharma.com. Forward Looking Statements. federal securities laws.

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The Pharma Data

JANUARY 3, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. PHILADELPHIA, Jan. 04, 2021 (GLOBE NEWSWIRE) — Passage Bio , Inc. Forward-Looking Statements.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 26, 2020

It may also allow for priority or rolling review of a company’s Biologics License Application (BLA). All forward-looking statements contained in this press release speak only as of the date on which they were made. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

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The Pharma Data

JANUARY 5, 2021

Caregivers of these patients often prioritize hyperphagia, the most common genetic cause of life-threatening childhood obesity, and anxiety as the most important symptoms for treatment of the disease. PWS, an orphan disease, is a complex genetic disorder with clinical manifestations on the endocrine and neurological systems.

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The Pharma Data

JANUARY 27, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. PHILADELPHIA, Jan. 28, 2021 (GLOBE NEWSWIRE) — Passage Bio , Inc. More information is available at www.passagebio.com. Forward-Looking Statements.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 30, 2020

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

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Production Antibody Manufacturing Trials 52

The Pharma Data

NOVEMBER 20, 2020

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. Eiger licensed exclusive worldwide rights to lonafarnib from Merck, known as MSD outside of the United States and Canada. ABOUT THE PROGERIA RESEARCH FOUNDATION.

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FDA Approval Genetics Clinical Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 8, 2020

Novavax is a leading innovator of recombinant vaccines; its proprietary recombinant technology platform combines the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles in order to address urgent global health needs. Sanofi, Empowering Life. For further information, please visit www.sanofi.com.

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The Pharma Data

JULY 27, 2021

This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. Amgen Forward-Looking Statements This news release contains forward-looking statements that are based on the current expectations and beliefs of Amgen. About Teneobio Teneobio, Inc.

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Production Antibody Sales Development 52

The Pharma Data

APRIL 18, 2022

Novavax licensed and transferred its manufacturing technologies to enable Takeda to develop and manufacture the vaccine at its facility in Hikari. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience and Gastroenterology (GI).

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Vaccination Vaccine Manufacturing Clinical Trials 52

The Pharma Data

APRIL 8, 2022

Based on these studies, one of those drugs was licensed to Cantex Pharmaceuticals for the treatment of COVID-19 and other inflammatory lung diseases. They also identified several drugs that reduced the production of inflammatory cytokines in infected Alveolus Chips, which could be useful in treating excessive inflammation in the lung.

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The Pharma Data

SEPTEMBER 8, 2020

is a leading innovator of recombinant vaccines; its proprietary recombinant technology platform combines the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles in order to address urgent global health needs. This press release contains “forward-looking statements” of. www.novavax.com.

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Vaccination Vaccine Containment Clinical Trials 52

The Pharma Data

SEPTEMBER 20, 2020

About KRAS The RAS gene family, which has been the subject of almost four decades of research, contains some of the most frequently mutated oncogenes in human cancers. To learn more about Amgen ‘s innovative pipeline with diverse modalities and genetically validated targets, please visit AmgenOncology.com.

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The Pharma Data

OCTOBER 18, 2020

This press release contains forward-looking statements of AVEO within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements.

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The Pharma Data

SEPTEMBER 16, 2020

To learn more about Amgen’s innovative pipeline with diverse modalities and genetically validated targets, please visit www.AmgenOncology.com. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Production 52

Production Sales Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 30, 2021

Starting with an initial dose 8 , Leqvio was then administered again at three months and then every six months thereafter in 482 participants with clinical or genetic evidence of heterozygous familial hypercholesterolemia and elevated LDL-C, despite a maximally tolerated dose of LDL-C-lowering therapies (e.g., a statin or ezetimibe).

RNA 52

RNA Trials Production Containment 52

The Pharma Data

OCTOBER 22, 2020

Genetic and preclinical experiments suggest that ADCC can be enhanced by NK-stimulators. Innate Pharma’s commercial-stage product, Lumoxiti, in-licensed from AstraZeneca in the US, EU and Switzerland, was approved by the FDA in September 2018. About Cetuximab: Cetuximab is an anti-EGFR monoclonal antibody.

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Clinical Trials Clinical Trials Trials Antibody 40

The Pharma Data

OCTOBER 14, 2020

For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.

FDA Approval 52

FDA Approval Antibody Production Trials 52

The Pharma Data

NOVEMBER 20, 2020

The EUA is temporary and does not take the place of a formal biologics license application (BLA) submission review and approval process. The FDA grants Emergency Use Authorization to medicines that may help diagnose, treat or prevent a life-threatening disease when adequate and approved alternatives are not available.

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Antibody Production Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 11, 2020

Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a biologics license application (BLA) for a rare pediatric disease product application may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product. .

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The Pharma Data

NOVEMBER 10, 2020

Painless is the company’s research platform in the field of pain and contains two projects: ACD440, which is a clinical candidate for the treatment of neuropathic pain, and TrkA-NAM, which targets severe pain in conditions such as osteoarthritis. The main risk factors to develop Alzheimer’s are age and genetic causes.

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