BioPharma Drive: Drug Pricing

AUGUST 28, 2023

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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The Pharma Data

JANUARY 19, 2021

Estrogen Therapy Need Not Stop for Gender-Affirming Surgery. 19, 2021 — For transgender and gender nonbinary (TGNB) patients undergoing primary vaginoplasty, perioperative venous thromboembolism (VTE) occurs rarely, regardless of whether hormone therapy is suspended prior to surgery, according to a study published online Jan.

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The Pharma Data

JUNE 2, 2022

New multi-program collaboration to develop allogeneic TCR-T/CAR-T programs brings together Immatics’ allogeneic gamma delta T cell therapy platform ACTallo ® with Bristol Myers Squibb’s technologies and oncology drug development expertise. Chief Executive Officer and Co-Founder of Immatics. “We FRCP, D.Phil.,

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The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

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The Pharma Data

JANUARY 25, 2021

a biotechnology company developing cell and gene therapy treatments for patients suffering from vascular disease, today announced that it raised up to $5.5M that will lead clinical testing of a novel gene therapy for a serious vascular disease in Europe. The company is a 2019 spin-out of the University of Miami co-founded by Robert L.

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Broad Institute

JULY 10, 2024

A three-drug cocktail known as Trikafta has greatly improved patient quality of life since its development in 2019, but can cause cataracts and liver damage and must be taken daily at a cost of about $300,000 per year. The most common is a three base-pair CTT deletion that causes the ion channel protein to misfold and degrade.

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The Pharma Data

APRIL 19, 2022

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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The Pharma Data

MARCH 25, 2021

this week announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma 1 and R/R follicular lymphoma. Bristol Myers Squibb K.K.

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The Pharma Data

JULY 2, 2023

The new approach in cancer therapy with innovative mechanism-of-action for ferroptosis induction A team of researchers led by Dr. Marcus Conrad from Helmholtz Munich discovered a novel anti-cancer drug, called icFSP1, which sensitizes cancer cells to ferroptosis.

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The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Mutations in OTOF lead to severe or profound congenital hearing loss. About Sensorion.

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Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. It’s called Roctavian and it’s only available for people who don’t have antibodies against AAV5, which is the viral vector used to deliver the gene therapy. From Moazami et al.

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Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. It’s called Roctavian and it’s only available for people who don’t have antibodies against AAV5, which is the viral vector used to deliver the gene therapy. From Moazami et al.

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The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Photo courtesy of Bayer.

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Antidote

JULY 29, 2022

billion to bring a new therapy to market. In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S.

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The Pharma Data

DECEMBER 21, 2020

The FDA granted cilta-cel Breakthrough Therapy and Orphan Drug designations in 2019, and the therapy received a PRIME Priority Medicines designation from the European Commission (EC) that same year and an Orphan Drug designation from the EC in February 2020. Source link.

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The Pharma Data

AUGUST 4, 2020

Therapy has been approved to treat advanced NTRK fusion-positive solid tumours. The European Commission (EC) has granted conditional marketing authorisation for Rozlytrek (entrectinib) for the treatment of advanced solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion.

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The Pharma Data

NOVEMBER 1, 2020

Corlieve’s lead program employs a novel AAV gene therapy approach for the treatment of refractory temporal lobe epilepsy (TLE), the most commonly diagnosed focal epilepsy in humans. Maryland , US), a leading gene therapy company. Maryland , US), a leading gene therapy company. PARIS , Nov.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Drug Discovery Today

DECEMBER 17, 2019

December 10, 2019 — Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, today announced that it has completed clinical production of Bridge Therapeutics Inc.’s Somerset, N.J. Birmingham, Ala.–

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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The Pharma Data

OCTOBER 27, 2020

Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. at Ophthalmic Consultants of Boston. About FOCUS.

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Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). Nature 578 , 229-236 (2020). e419 (2023).

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The Pharma Data

DECEMBER 2, 2020

Based on the Phase 2 study findings to date, marstacimab may have the potential to offer improved bleed control via subcutaneous injection and potentially eliminate the need for prophylactic factor replacement, providing an enhanced treatment option compared to factor replacement therapy.”. In September 2019, the U.S.

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DrugBank

OCTOBER 4, 2024

The FDA's accelerated approval pathways, such as the Breakthrough Therapy designation and the Priority Review program, have shortened development timelines and reduced costs for certain drugs, making them more attractive acquisition targets. However, such acquisitions also entail significant risks for biotech firms.

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Drug Target Review

OCTOBER 4, 2023

Dual payload ADCs As effective as therapies have been in treating solid and haematological cancers, tumour heterogeneity and resistance remain major clinical challenges. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution. Cancers 11 , 394 (2019).

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Common Sense for Drug Policy Blog

JUNE 6, 2024

2018 ; Llewellyn and Tober 2010; Denham 2019 ). 2019 ), who found that their sample’s consumption ‘transcended IPEDs to encompass a much broader cocktail of substances’ (Salinas et al. 2019 : 49) including recreational drugs like cannabis and cocaine (see also Turnock 2021b ). 2014 ; Begley et al. Fincoeur et al.

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The Pharma Data

JANUARY 21, 2021

They all underwent transplants at various Italian medical centers between 1997 and 2019. For one, the patients were not part of a clinical trial that directly tested stem cell transplants against standard MS medications. So it’s unclear exactly how such transplants measure up against the most effective MS drugs now available.

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The Pharma Data

MAY 4, 2021

INDIANAPOLIS , May 4, 2021 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) is donating COVID-19 therapies to Direct Relief, enabling the humanitarian organization to provide COVID-19 therapies at no cost to low- and lower-middle-income countries most heavily impacted by the pandemic.

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The Pharma Data

FEBRUARY 22, 2021

Efanesoctocog alfa, a novel and investigational factor VIII therapy independent of von Willebrand Factor, is designed to provide near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen. In 2019, Sobi’s revenues amounted to SEK 14.2

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The Pharma Data

NOVEMBER 20, 2020

NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. TARRYTOWN, N.Y., November 21, 2020 – Regeneron Pharmaceuticals, Inc.

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Broad Institute

NOVEMBER 6, 2024

This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor. Carol’s breast cancer diagnosis and related immunotherapy treatment brought her to the SIC Service at MGH in 2019, where she stayed for two weeks after developing myocarditis. “I

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The Pharma Data

NOVEMBER 19, 2020

19, 2020 — The volume of transcatheter aortic valve replacement (TAVR) procedures in the United States increased from 2011 to 2019, exceeding surgical aortic valve replacements in 2019, and 30-day mortality and stroke rates have decreased since 2011, according to a study published in the Nov. THURSDAY, Nov. Carroll, M.D.,

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Drug Target Review

DECEMBER 12, 2023

He was the founding CEO since the inception of Araris in 2019 securing $40M in financing from Swiss, UK, US and Korean investors and $2.5M He positioned Araris to shift the paradigm in how to think about developing targeted cancer therapies and since October 2023, he transitioned to become its CSO.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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