Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9

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Gene Therapy Gene Manufacturing Medicine 52

Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

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Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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Gene Editing Gene In-Vivo Gene Therapy 147

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

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pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.

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Gene Editing In-Vivo Gene Sequencing Gene 88

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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Drug Discovery World

APRIL 15, 2024

Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types. In addition, I believe we will see more exosome-based therapies enter the clinic in 2024.

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Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Pharma Marketing Network

DECEMBER 21, 2020

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. Genetic literacy among clinicians may not be as high as you think.

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Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

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Gene Editing Gene Gene Therapy Drugs 59

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 59

Drug Discovery World

APRIL 3, 2023

DS: What motivated you and Dr David Bunka, CTO, to create Aptamer and did you imagine you would one day be operating on such a global scale?    I quickly recognised the commercial value of aptamers as being able to address the gap in the market where antibodies fail to perform, and together we established the business to begin aptamer development.

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Pharmaceutical Technology

JULY 20, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes.

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Gene Therapy Gene Development In-Vivo 147

The Pharma Data

NOVEMBER 22, 2020

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years’ experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. MILAN, Italy and NEW YORK, Nov. About Genenta Science.

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Gene Therapy Gene In-Vivo Clinical Trials 40

pharmaphorum

MAY 27, 2021

Life sciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Funds will be used to expand the company’s presence across Asia and create an innovation hub in Boston for cell and gene therapy technologies.

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Life Science In-Vivo Manufacturing Gene Therapy 59

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

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Gene Editing Gene In-Vivo Genetics 52

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Already in markets in China, BeiGene’s Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc? and Europe.

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XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

Pharmaceutical Technology

AUGUST 18, 2022

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. An ex-vivo lentiviral vector (LVV) gene therapy, Zynteglo’s every dose is made by genetically modifying the bone marrow stem cells of the patient to produce functional beta-globin.

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Gene Therapy Gene Genotype Genetics 246

Roots Analysis

JANUARY 14, 2024

Moreover, the exuberant development of biologics has revolutionized the treatment of a range of therapeutic conditions, which has further contributed to the exponential growth in the current demand for biologic therapies developed by biologics manufacturing companies.

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Contract Manufacturing Manufacturing Protein Bacteria 40

Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

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Pharmaceutical Technology

SEPTEMBER 19, 2022

A one-time gene therapy, Skysona leverages ex-vivo transduction with the Lenti-D lentiviral vector for adding the ABCD1 gene’s functional copies into the hematopoietic stem cells of the patient. The treatment is indicated for usage in boys aged four to 17 years with CALD. 1) in Skysona-treated and untreated subjects.

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FDA Approval In-Vivo Gene Therapy Clinical Trials 246

Drug Discovery World

SEPTEMBER 28, 2022

Yet despite this, the biggest players in the global pharma sector are increasingly opening their doors to partnerships with AI and machine learning companies, cognisant of the potential benefits these could provide across broad therapy areas such as oncology, autoimmune and rare diseases. . The simple answer is no.

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Pharma Companies Drugs In-Vivo Genetics 52

Delveinsight

JANUARY 18, 2021

Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father. The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. .

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Marketing Gene Drugs Protein 52

World of DTC Marketing

JANUARY 31, 2021

Smaller biotechs that are developing new drugs usually don’t have the resources to take new drugs to market. The cost of bringing a drug to market decreased from $2,168 million in 2018 to $1,981 in 2019. Still, she is missing a lot of facts at a time when hospital and health insurance costs are soaring. percent from 2018.

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XTalks

DECEMBER 20, 2023

In 2024, pharma companies and emerging biotechs will improve candidate safety profiles and reduce off-target effects by exploiting predictive intelligence unlocked by AI,” she predicts. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

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Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

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Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

NOVEMBER 10, 2020

This model has the benefit of giving companies direct oversight of their in vivo studies, allowing them to move faster while operationalizing every dollar of their seed financing. Photo courtesy of Mispro. There’s an emerging business model in biotech town, and it’s called the Contract Vivarium (CV).

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Marketing In-Vivo Scientist Research 52

Drug Discovery World

APRIL 12, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

OCTOBER 17, 2022

By Dr Christof Gänzler, Product Marketing Biology at PerkinElmer Informatics. . In today’s quest to accelerate time to market for novel drugs, part of the success formula requires leveraging discovery data with more precision and speed, and earlier in the process. It takes more than speed to advance and maintain performance.

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Marketing Scientist Gene Expression Drugs 52

Drug Discovery World

JANUARY 20, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

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Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

NOVEMBER 7, 2023

MT: What are the challenges you face in taking research to market, and how have you begun to address them? RB: As a CRO, we aren’t bringing our research to market per se, but we are instrumental in helping other organisations assess test articles in their development pipeline at various stages. Every day one learns something.

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Research In-Vivo Gene Therapy Development 59

Drug Discovery World

OCTOBER 13, 2022

As with mRNA technologies, lipid nanoparticle carriers, have seen similar development, advancing the potential of mRNA and enabling efficient in vivo delivery and potentially prolonged antigen expression 4. Why mRNA has potential . One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited.

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Drugs Manufacturing Vaccination Vaccine 52

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

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Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

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Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

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