Pharmaceutical Technology

JUNE 29, 2022

The pharmaceutical industry often relies on enzymes to perform a process known as biocatalysis, which facilitates the cost-effective and sustainable production of small molecule drugs at scale. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior.

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XTalks

AUGUST 21, 2023

The US Food and Drug Administration (FDA) has approved Ipsen’s groundbreaking Sohonos (palovarotene) capsules for the treatment of fibrodysplasia ossificans progressive (FOP), an ultra-rare bone disease. Sohonos, the first and only approved drug, offers hope to those battling the disease.

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XTalks

MAY 17, 2024

Xtalks spoke with Richard Philipson, MD, Chief Medical Officer (CMO) at Calliditas Therapeutics, a Sweden-based company with full commercial operations in the US focused on developing treatments for rare diseases to learn more about the continuing evolution of rare disease drug development.

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World of DTC Marketing

JANUARY 1, 2022

1ne: The value proposition of prescription drugs still outweighs potential side effects – Wegovy’s demand quickly exceeded supply after the FDA approved once-weekly semaglutide injections. People don’t want to lose weight through diet and exercise if they can lose it through a prescription drug.

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pharmaphorum

JUNE 24, 2022

Last year, AstraZeneca reported that a new drug to treat Wilson disease developed by its Alexion rare diseases unit had hit the mark in a phase 3 trial, setting it on course for regulatory filings. The post Data backs AZ’s Wilson disease drug, but with a question mark appeared first on.

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STAT News

MARCH 6, 2023

Still, when doctors told her last year that the cancer was growing despite two operations, radiation therapy, and a fifth regimen of chemotherapy, the retired business-meeting facilitator decided to do something unorthodox: spend $83,000 out of pocket on an unproven experimental cancer vaccine.

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pharmaphorum

AUGUST 25, 2022

That was until my doctors stunned me with the news that I had cirrhosis caused by non-alcoholic steatohepatitis, or NASH, a severe form of fatty liver disease that often has few or no symptoms until very late stages. Obesity, diabetes, and genetics are the primary drivers, and COVID is about to make this exponentially worse.

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Bioengineer

JULY 22, 2021

Geneticists from Trinity College Dublin have discovered how a specific genetic mutation called H3K27M causes a devastating, incurable childhood cancer, known as diffuse midline glioma (DMG), and – in lab studies working with model cell types – successfully reverse its effects to slow cancer cell growth with a targeted drug.

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Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

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The Pharma Data

AUGUST 16, 2021

If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. At full stretch, researchers are developing various vaccines and drugs – with different degrees of success. Molnupiravir, another antiviral drug candidate, was originally developed to treat influenza.

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Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . We used these peptides to act as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye.” .

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The Pharma Data

MAY 9, 2022

No drugs are approved for these aggressive tumors, which occur in up to 20% of cases and can lead to patient disability or even death. The drug is a newer cancer treatment called abemaciclib. Currently, after surgery, doctors examine a specimen of a tumor under a microscope and grade it one, two or three in its aggressiveness.

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The Pharma Data

DECEMBER 28, 2020

The Corps is a group of doctors, public health professionals and community advocates working to reverse the COVID-19 outbreak and protect our city. About Fulgent Genetics. TEMPLE CITY, Calif., It ensures that anyone with the virus receives care and can safely isolate to prevent the spread. For more information, visit [link].

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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pharmaphorum

DECEMBER 21, 2021

However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 proved to be a remarkable year for orphan drug approvals, with the US FDA approving more orphan designated treatments than non-orphan drugs. Headway being made.

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The Pharma Data

SEPTEMBER 1, 2021

Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. The approach allows those cells to remain in the body for a long time and secrete a drug whenever there is a flare of inflammation.” million adults in the United States.

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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. The disease is also difficult to diagnose and often takes around six years before doctors correctly identify it.

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pharmaphorum

AUGUST 11, 2022

More than 60 percent of all American adults take prescription drugs , amounting to approximately 131 million individuals. These drugs have been rigorously tested by regulatory bodies around the world before they’re made available to ensure they work as labelled, but despite that, adverse events crop up.

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Drug Discovery World

NOVEMBER 27, 2023

Across the biotherapeutics workflow The landscape of large molecule drug development has evolved significantly in recent decades. Prioritising critical attributes in the upstream workflow is crucial for predicting and preventing challenges downstream, ultimately avoiding high development costs and delays in drug approvals.

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pharmaphorum

AUGUST 13, 2020

Exon-skipping drugs are used to patch the mutations and allow the gene to produce partially functional dystrophin. Viltepso can be used to treat around 8% of DMD patients and previously approved DMD drugs has not been shown to change the course of the disease. In the study, dystrophin levels increased, on average, from 0.6%

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pharmaphorum

OCTOBER 25, 2021

The company is best known for its saliva-based health and ancestry DNA testing targeted at consumers, but also uses its genetic database to look for new targets for drug development. The transaction is expected to close before the end of the year.

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pharmaphorum

APRIL 8, 2021

COVID-19 has been a trial-by-fire showing just how quickly drugs and vaccines can be developed under the right circumstances – but there is much to do to ensure this progress is not lost, said speakers at WIRED Health 2021, including the CEOs of BioNTech and BenevolentAI. . We published our hypothesis in the Lancet on February 4 in 2020.

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XTalks

NOVEMBER 18, 2021

“The first time I heard the term hypertrophic cardiomyopathy, I was in the doctor’s office in the room getting diagnosed. HCM is a genetic heart condition in which the muscle of the heart thickens, causing it to work harder to pump blood. Through genetic testing, it was revealed that Juanea carries the gene for the heart condition.

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FDA Law Blog

MARCH 13, 2023

Bauer, Senior Regulatory Drug Expert & James E. Valentine — Incorporating patient and caregiver experiences into every phase of drug development has become increasingly prioritized during both development and review ( see, e.g. , previous coverage here ). By Larry J. There have been challenges in gene therapy development.

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STAT News

APRIL 3, 2023

Conner’s doctors expect that this groundbreaking gene therapy, which costs a staggering $3 million a patient, will stave off a fatal degenerative brain disease and save his life. Millions of stem cells that were collected from 6-year-old Conner Hess’s blood in January flowed through the IV and entered his bloodstream.

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pharmaphorum

FEBRUARY 25, 2021

Chaired by the Genetic Alliance, the virtual meeting saw representatives from Scotland, England, Wales, and Northern Ireland discuss the implementation of the UK Government’s Rare Disease Framework. We now know that 80% of rare diseases have a genetic origin. Genomic technology, therefore, has a key role to play in our work,” he said.

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The Pharma Data

DECEMBER 17, 2020

7, 2020 — After starting a drug that’s officially approved to treat a type of blood cancer, a young man with type 1 diabetes was able to stop using insulin. Dr. Lisa Forbes — his doctor and co-author of a letter describing his case in the Oct. This patient had a genetic mutation that ruxolitinib is known to work on.

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pharmaphorum

JANUARY 15, 2023

Neurological conditions like Multiple Sclerosis (MS) are an incredibly important area of research when it comes to drug discovery, but under that (sometimes mistaken) umbrella diagnosis occur other, rarer diseases, such as Neuromyelitis Optica Spectrum Disorder (NMOSD). Until recently, NMOSD was regarded as an optic-spinal form of MS.

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pharmaphorum

SEPTEMBER 21, 2021

“We need to ensure qualified patients are diagnosed, identified and engaged early on in drug development”. Doctors are often unfamiliar with these conditions. Doctors are heavily reliant on the observations of parents or caregivers and are constrained by the ability of the patient to convey symptoms they are experiencing.

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pharmaphorum

OCTOBER 6, 2022

These include exposure to various chemicals associated with the war, from chemical warfare agents to pyridostigmine bromide – a preventative drug given to soldiers at risk of exposure to toxic chemicals. Not only does this make drug discovery more efficient, but it is also less resource intensive. Tinker, tailor, soldier, therapy.

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pharmaphorum

DECEMBER 21, 2020

As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein. They are very good doctors, scientists and entrepreneurs. He told pharmaphorum: “They are very good doctors, scientists and entrepreneurs.

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pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

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pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans. The three drugs required intravenous dosing once a week and work only in around a third of DMD patients.

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Drug Discovery World

SEPTEMBER 5, 2022

An experimental combination of two drugs halts the progression of small cell lung cancer, the deadliest form of lung cancer, according to a recent study. One of the drugs, cyclophosphamide, was once used to treat small cell lung cancer, but was displaced in favour of platinum-based drugs in the 1980s.

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XTalks

APRIL 6, 2022

If that happens, it is a big setback for patients and the doctors treating them,” said Dr. Rosenfeld in a news release by Cancer Research UK. We’re hoping that this technology could help doctors decide when additional rounds of treatment are needed, and could save lives.”.

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pharmaphorum

JANUARY 6, 2021

It’s likely no coincidence that Pfizer’s flagship project in 2020 while the design of the logo was being finalised was unquestionably its high-profile alliance with BioNTech on the RNA-based coronavirus vaccine Comirnaty, which provided further evidence of the potential of harnessing genetic toolkits to tackle health issues.

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XTalks

JUNE 30, 2023

The US Food and Drug Administration (FDA) approved BioMarin’s Roctavian, an adeno-associated virus (AAV) vector-based gene therapy, for the treatment of adults with severe hemophilia A. 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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