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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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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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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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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Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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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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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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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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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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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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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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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

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

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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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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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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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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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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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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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

MARCH 3, 2025

In this interview, Professor Schwamborn discusses his research, the promise of brain organoids, and his vision for the future of stem cell-based therapies. ” By using personalised models, researchers can identify subgroups of patients with similar molecular characteristics, potentially leading to the development of targeted therapies.

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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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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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Drug Target Review

SEPTEMBER 4, 2023

7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria. 2019): CRISPR in Parasitology: Not Exactly Cut and Dried! 2019): Biological plasticity rescues target activity in CRISPR knockouts, Nature Methods.

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Broad Institute

NOVEMBER 13, 2023

This commitment to the Gerstner Center for Cancer Diagnostics includes $10 million for current support for the Center and $10 million in endowed funding, adding to the $15 million originally committed by Gerstner and an initial endowment of $10 million from the Eli & Edythe Broad Foundation to establish the Center in 2019.

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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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Eye on FDA

JULY 5, 2022

Most notably in 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed into law, bring to bear among other things, breakthrough therapy designation. by mid-year 2019, there were only 13 approvals of new entities but the year netted out with a respectable 48.

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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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Broad Institute

JANUARY 8, 2024

Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing. DOI: 10.1038/s41587-023-02078-y Tags: Liu Lab Gene editing Rare Disease Gene-based therapy delivery Liu is also a Howard Hughes Medical Institute investigator and a professor at Harvard University.

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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

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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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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Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. 2019 May 22;10:1078. Harjunpää H, Llort Asens M, Guenther C, Fagerholm SC.

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Drug Target Review

OCTOBER 12, 2023

In turn this will predict disease risks and develop more effective therapies. 2019 August 30 [cited 2023 October 6]; 11(233):20. Looking ahead, Li, Shen and Gan labs will use more complex model systems that mimic the human brain, like human cerebral organoids, to further the analysis of AD risk variants. Frontiers [Internet].

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