Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

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Pharmaceutical Technology

APRIL 26, 2023

BioMarin senior vice-president and chief scientific officer Kevin Eggan stated: “Activating RNA therapeutics have the potential to change the way we treat certain genetic diseases, particularly those characterised by the limited production of key proteins. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets. About Novartis.

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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna Genomics general manager and chief scientific officer Eric Huang said: “Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA.”

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Pharmaceutical Technology

AUGUST 24, 2022

Mode rna has submitted an application to the US Food and Drug Administration (FDA) to obtain emergency use authorization (EUA) for mRNA-1273.222, its BA.4/BA.5 Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 5 Omicron-targeting bivalent booster vaccine for Covid-19.

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The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. ” The Institute will be headquartered in 334,000 sq.

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Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Genetics Medicine Gene Therapy Marketing 52

XTalks

OCTOBER 26, 2021

Givlaari was approved by the European Medicines Agency (EMA) in March 2020 and by the US Food and Drug Administration ( FDA ) in 2019. Related: Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA. This is a huge development for patients.”.

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XTalks

JANUARY 8, 2024

Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. Getting a drug to market used to be a relatively simple and straightforward affair. That’s just not the case anymore.

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Clinical Trials Clinical Trials Trials Gene Therapy 105

Scienmag

APRIL 21, 2021

Targeting an RNA to interrupt its activity could improve patient response to therapy and increase their overall survival. […]. Credit: Yutaka Kondo Nagoya University researchers and colleagues in Japan have uncovered a molecular pathway that enhances chemotherapy resistance in some pancreatic cancer patients.

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Delveinsight

SEPTEMBER 14, 2021

ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B. Simeon George, M.D.,

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Pharmaceutical Technology

APRIL 24, 2023

Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. This intersection will be remembered as one of the most significant achievements in science and medicine”. Langer, now a David H.

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Drug Delivery Vaccine Vaccination Research 147

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

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Marketing Gene Sequencing DNA Genomics 52

pharmaphorum

AUGUST 5, 2021

Bayer headlined its second-quarter results this morning by unveiling a deal to buy Vividion Therapeutics, saying it will boost its ability to develop medicines against targets once considered “undruggable.” ” Bayer is paying $1.5 billion ($5.3

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pharmaphorum

JULY 21, 2021

Leqvio – acquired via Novartis’ near $10 billion acquisition of the Medicine Company in 2019 – is already approved in Europe but getting it on the US market is central to Novartis’ hopes of blockbuster sales for the product. Its innovative medicines division climbed 15% to $10.6

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Sales Gene Therapy RNA Production 52

The Pharma Data

MARCH 5, 2021

This will be adapted to the needs of messenger RNA vaccine production for CureVac’s CVnCoV, the manufacturing of which is highly complex. Novartis has also announced a collaboration with Molecular Partners to develop two DARPin® therapies designed for potential use against COVID-19.

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Manufacturing Vaccine Vaccination Production 52

The Pharma Data

DECEMBER 13, 2020

Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. Before that, he was Director of Development at Cobalt Biomedicine leading CAR-T and other cell/gene therapy programs, and R&D Director at OvaScience developing stem cell-based products.

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Business Development Development Gene Editing In-Vivo 40

pharmaphorum

APRIL 28, 2022

These included setting their sights beyond haemophilia and growth hormone deficiencies into the wider, but still focused, spaces of red blood and endocrine disorders; a commercial enhancement program helping the company to launch medicines faster and with more impact; and a number of acquisitions that have given the company footholds in new treatment (..)

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XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. Targeting the CNS directly has been successful in modern medicine. Next Steps for Targeted CNS Delivery.

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Trials Drug Delivery Drugs Gene 83

Pharmaceutical Technology

OCTOBER 11, 2022

Biotechnology company Ochre Bio has raised $30m in a Series A financing round to develop RNA therapies for chronic liver diseases. It also intends to turn the insights from its research into RNA-based drug candidates, and they will be tested in human livers at the company’s recently opened ‘Liver ICUs’ in the US.

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Pharmaceutical Technology

APRIL 27, 2023

Orbital Therapeutics has raised $270m in a Series A round led by ARCH Venture Partners to advance a portfolio of programmable RNA therapeutics. Orbital will use the new funding to increase the application of RNA-based medicines for use in the fields of new vaccines, immunomodulation and protein replacement.

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Pharmaceutical Technology

APRIL 20, 2023

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

JUNE 1, 2023

A new intracellular drug delivery centre will be established in the UK to support potential ribonucleic acid (RNA) vaccines and therapeutics , as well as the development of innovative drug delivery technologies. The funding is being provided under the agency’s Transforming Medicines Manufacturing programme.

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Drug Delivery RNA Development Drugs 242

pharmaphorum

OCTOBER 12, 2022

We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says. Genomic medicine is a rapidly growing space in which researchers use the information in a person’s genes to identify and administer medical treatment.”.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. 5 in the New England Journal of Medicine.

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Gene Therapy Gene Gene Editing Genetics 52

Pharmaceutical Technology

FEBRUARY 1, 2023

Moderna brings up the rear after announcing Phase III results with its own messenger RNA (mRNA) RSV vaccine earlier this month. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.

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pharmaphorum

AUGUST 22, 2022

“We’re looking at how healthcare is significantly changing the way that we understand, map, and then build medicines and how that can change by making health more operationally efficient,” Torrance states. The status quo in the healthcare sector is an antiquated and fossilised model, Torrance says.

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The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Clinical Development Gene Therapy Development Gene 52

XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

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Life Science Gene Therapy DNA Vaccine 105

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

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Pharmaceutical Technology

APRIL 27, 2023

Genomic medicine development was ranked as the top industry trend for 2023 in a survey of 198 GlobalData Pharma clients and prospects conducted between October and November 2022. GlobalData pharmaceutical analysts project that RNA-based gene therapies for oncology will grow from zero in 2022 to $4.6 billion by 2028.

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Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. All our equipment is in line with the latest European Medicines Agency (EMA) and FDA methods. Ribonucleic acid (RNA) based technologies are among many innovative approaches explored to tackle the Covid-19 pandemic.

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Delveinsight

AUGUST 5, 2021

The acquisition is the recent sign that large pharmaceutical companies view messenger RNA, which BioNTech and Moderna utilized to develop the COVID-19 vaccines now cleared for use in dozens of countries, as a crucial drug-making platform. Fibroblasts are part of the platform fueling cell-based gene therapy biotech Castle Creek.

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Vaccine Vaccination Gene Therapy Development 98

Worldwide Clinical Trials

MAY 30, 2024

was withdrawn, rendering it unapproved for patient use, based on negative review comments by the Committee for Medicinal Products for Human Use. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient. The MAA file in the E.U.

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Pharmaceutical Technology

FEBRUARY 20, 2023

He says these issues must be urgently addressed over the next few years as advancing medical knowledge and the role of genomics in treating disease ushers in a new era of precision medicine and new potential to address diseases that affect certain populations. The technology platforms are almost the same.

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pharmaphorum

JANUARY 6, 2022

Medicine budget constraint, structural fund largesse. The period 2021-2025 was always going to be one of relatively low loss of exclusivity cost relief, with 18% of, combined with unprecedented levels of COVID-19 related spend, medicines budgets will be challenged for years to come. RNA Therapeutics come to the fore.

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XTalks

AUGUST 2, 2023

With each passing year, pharmaceutical companies around the globe strive to deliver cutting-edge medicines, therapies and vaccines that impact the lives of millions. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. But all three drugs have new competitors.

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