Drug Target Review

MARCH 21, 2024

Dystrophin is mutated in Duchenne muscular dystrophy (DMD), a rare disease that causes progressive muscle weakness and atrophy. There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. 2020 Jul 3;10(1):10967. Scientific reports.

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Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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Drug Target Review

NOVEMBER 7, 2023

To target proteins involved in causing or spreading disease, typically a drug will obstruct a protein’s active site so it cannot function. Proteins can become part of a cancerous tumour and allow for its unrestricted growth, cause diseases like Alzheimer’s , and build up in the heart to impact how it pumps blood around the body.

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The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced. To date, a total of 19 trial participants have been dosed with the gene therapy.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) granted Breakthrough Therapy designation for donanemab, Eli Lilly and Company’s (NYSE: LLY) investigational antibody therapy for Alzheimer’s disease (AD). Dementia due to Alzheimer’s disease is the most common form of dementia, accounting for 60 to 80 percent of all cases 1.

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The Pharma Data

OCTOBER 21, 2020

22, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the company will host a conference call and live webcast on Thursday, November 5, 2020 at 8:00 a.m. CAMBRIDGE, Mass.,

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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The Pharma Data

NOVEMBER 18, 2020

Per the deal, Obsidian Therapeutics will provide its cytoDRiVE delivery platform to the university to speed research into TILs, which target tumors by reversing the expression of certain cancer-causing proteins via an oral therapy. Source link.

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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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The Pharma Data

NOVEMBER 8, 2020

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs. LONDON and RALEIGH, N.C., For more information, please visit www.veronapharma.com. Forward-Looking Statements.

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The Pharma Data

NOVEMBER 18, 2020

Antimicrobial resistance occurs when microorganisms resist the effects of medications, making infections harder to treat and increasing the risk of disease spread, severe illness and death. Key definitions. Combatting antimicrobial resistance is a global. efforts to address antimicrobial resistance. Department information note. infants. .

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The Pharma Data

DECEMBER 30, 2020

The year 2020 will certainly go down in history as the year of COVID-19. Gilead Sciences Makes Multiple Bets : When Gilead Sciences wasn’t working on remdesivir in COVID-19, the company seemed to be constantly flexing its M&A muscle over the course of 2020. It began in May with the $4.9

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The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

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New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. 2020-01-02. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. J Pers Med.

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The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. The researchers said the technique could break the cycle of disease passed from mother to baby through mutations in mitochondrial DNA (mtDNA). FRIDAY, Dec.

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The Pharma Data

OCTOBER 20, 2020

Kidney Disease, Injury Linked to Higher Mortality With COVID-19. 20, 2020 — Renal impairment in patients admitted to intensive care with COVID-19 is common and is associated with high mortality, according to a study published online Oct. © 2020 HealthDay. Posted: October 2020. Professional. TUESDAY, Oct.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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The Pharma Data

NOVEMBER 20, 2020

20, 2020 — Hormone therapy can be a lifesaver for men with prostate cancer , but it also appears to put some at increased risk of heart problems, a new study reports. At the start of the study, 4 out of 5 men had two or more risk factors for heart disease , the study says. FRIDAY, Nov.

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

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The Pharma Data

OCTOBER 26, 2020

27, 2020 11:00 UTC. DALLAS–( BUSINESS WIRE )– Taysha Gene Therapies Inc. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S.

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The Pharma Data

APRIL 22, 2023

Multiple sclerosis (MS) is a devastating autoimmune disease that destroys the protective myelin covering around nerves, disrupting communication between the brain and body, and causing patients’ ability to move and function to progressively decline. million individuals that currently have to live with the disease.

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The Pharma Data

SEPTEMBER 16, 2020

16, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) will host two webcast calls for the investment community in conjunction with the European Society for Medical Oncology (ESMO) 2020 Virtual Congress. 20, 2020, at 11:00 a.m. THOUSAND OAKS, Calif. On Sunday, Sept. PDT, David M. Reese , M.D., Reese , M.D.,

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The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. 22, 2020 — Tagrisso ( osimertinib ) received approval as an adjuvant treatment for patients with non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, the U.S. © 2020 HealthDay. Professional.

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The Pharma Data

JULY 28, 2021

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa, a long-term enzyme replacement therapy for the treatment of people with Pompe disease. Avalglucosidase alfa is also currently undergoing review in other countries including the U.S.,

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The Pharma Data

SEPTEMBER 24, 2020

NYSE:PFE) declared a 38-cent fourth-quarter 2020 dividend on the company’s common stock, payable December 1, 2020, to holders of the Common Stock of record at the close of business on November 6, 2020. The fourth-quarter 2020 cash dividend will be the 328 th consecutive quarterly dividend paid by Pfizer. Pfizer Inc.:

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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The Pharma Data

SEPTEMBER 21, 2020

New data from two studies investigating the combination of Eisai’s multiple receptor tyrosine kinase inhibitor Lenvima (lenvatinib) and MSD’s blockbuster anti-PD-1 therapy Keytruda (pembrolizumab) were revealed at the European Society for Medical Oncology (ESMO) Virtual Congress 2020.

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The Pharma Data

DECEMBER 31, 2020

Monthly News Roundup – December 2020. In studies, the vaccines were found to be 94% to 95% effective in preventing COVID-19 disease. The partial piece of the spike protein cannot cause COVID-19 disease. Orladeyo: First Oral, Once-Daily Therapy to Prevent Hereditary Angioedema Attacks.

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The Pharma Data

JANUARY 18, 2021

“There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease,” said RA Session II, President, Founder and CEO of Taysha. “We Taysha’s commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.”.

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The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

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The Pharma Data

DECEMBER 18, 2020

18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).

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The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with neurodegenerative diseases for potential disease modification. Teva Pharmaceutical Industries Ltd.

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The Pharma Data

SEPTEMBER 28, 2020

Cardiovascular disease (CVD) is a general term for conditions affecting the heart or blood vessels. CVD has many causes: from smoking, diabetes, high blood pressure and obesity, to air pollution, genetic risk factors and less common conditions such as Chagas disease. Developing next-generation therapies.

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