Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Shilling was appointed to represent the company on the steering committee of the BGTC.

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BioSpace

SEPTEMBER 15, 2020

Clinical-stage biopharmaceutical firm ESCAPE Bio has snagged a $73 million crossover financing deal, led by Wellington Management Company, which will help advance the company’s two clinical development programs of precision neurology medicines for patients with genetic neurodegeneration.

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Worldwide Clinical Trials

FEBRUARY 6, 2023

When using genetic screening to identify clinical trial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article!

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Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

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Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

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Pharmaceutical Technology

MAY 19, 2023

ImmunoForge has expanded its licence agreement with Duke University in North Carolina, US, to jointly improve research and drug development opportunities. ImmunoForge will expedite the development of new drugs based on the elastin-like polypeptide (ELP) platform technology originally developed by Duke’s Professor Ashutosh Chilkoti.

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XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

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Pharmaceutical Technology

AUGUST 24, 2022

Lacerta Therapeutics has entered a new licensing and research partnership agreement with Eli Lilly and Company subsidiary, Prevail Therapeutics, to discover and develop adeno-associated virus (AAV) capsids for treating central nervous system (CNS) diseases. . Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

JUNE 6, 2023

Alkeus Pharmaceuticals has raised $150m in Series B financing to support the registration and launch of gildeuretinol (ALK-001) to treat Stargardt disease, a genetic cause of blindness in children and young adults. The genetic cause is well characterised, but to date there is no effective treatment.

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Drug Discovery World

APRIL 24, 2024

Evotec will identify best-in-class small molecules targeting a key fibrotic pathway with strong genetic support identified by Variant Bio and progress the programme towards the selection of a clinical development candidate(s) using its R&D platform.

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The Pharma Data

NOVEMBER 30, 2020

Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. About NVX-CoV2373 NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

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Drug Discovery Today

JULY 28, 2020

Cancer Research UK, the University of Southampton and Touchlight Genetics, a London based biotechnology company, today (Wednesday) announce a new clinical development partnership to progress a therapeutic DNA vaccine, TGL-100, into an early phase clinical trial targeting head and neck squamous cell carcinoma (HNSCC).

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Similarly, DNA molecules can be genetically modified and introduced into human cells, or individual patient cells can be genetically modified and reintroduced to the body. PK Planning for Gene Therapy Development Programs.

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Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

FEBRUARY 28, 2024

Lu Rahman selects some of the year’s interesting and noteworthy advances in cancer research drug discovery and development. As always, a year within cancer drug discovery and development brings with it significant breakthroughs, and 2023 has been no exception. Figures from the Amercian Cancer Society 1 state that “a little over 1.9

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Drug Discovery World

DECEMBER 19, 2022

Kite and Arcellx have entered a global strategic collaboration to co-develop and co-commercialise CART-ddBCMA for the treatment of patients with relapsed or refractory multiple myeloma. . The post Partnership to co-develop cell therapy for multiple myeloma appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

FEBRUARY 22, 2023

Taldefgrobep is a complete human anti-myostatin recombinant protein that is developed for lowering free myostatin. Biohaven Clinical Development vice-president Lindsey Lee Lair said: “We are very pleased the FDA granted Fast Track designation for taldefgrobep alfa for the treatment of SMA.

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Clinical Trials Clinical Trials Genetics Protein 246

Pharma Mirror

OCTOBER 26, 2021

a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options, has formed a new research collaboration with Mayo Clinic. Castle Creek Biosciences Inc., Deyle, M.D.,

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Outsourcing Pharma

AUGUST 31, 2021

According to patient-matching specialist TrialJectory, 90% of cancer patients know the vitality of genetic testing but more than half are not being tested.

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Genetics Clinical Development Development 69

Pharmaceutical Technology

MAY 17, 2023

Takeda rare genetics and haematology therapeutic area unit head Daniel Curran said: “There is a critical need for treatment options for people living with cTTP, an ultra-rare, life-threatening disorder that has no therapies specifically approved for prophylactic treatment.

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Pharmaceutical Technology

MAY 17, 2023

Boundless Bio intends to use the funds for the initial clinical development of the first ecDNA-directed therapy (ecDTx), BBI-355, through clinical data readouts from its ongoing Phase I/II POTENTIATE clinical trial. The company is developing the assay with SOPHiA GENETICS.

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. However, these complex treatments present challenges to developers over and above the well-documented hurdles of rare-disease research. “They called him ‘The Boy.’ Who wasn’t supposed to sit up.

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pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt. Vertex is also offering up to $1.3

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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.

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Pharmaceutical Technology

MARCH 1, 2023

The Covid-19 pandemic led to massive developments and scientific advances within the field of messenger ribonucleic acid (mRNA) vaccines. Pfizer /BioNTech and Moderna’s mRNA-based vaccines were the first to receive emergency use authorisation (EUA) out of all other Covid-19 vaccines under development in late 2020.

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Pharmaceutical Technology

DECEMBER 15, 2022

According to the research and development (R&D) deal, the combinatorial analytics platform of PrecisionLife will be applied to patient datasets to detect several novel therapeutic targets, which will be developed by Ono. .

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Pharmaceutical Technology

MARCH 2, 2023

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

Gene Editing 100

Gene Editing Genetic Engineering Engineer Clinical Trials 100

Pharmaceutical Technology

MARCH 2, 2023

QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Gene Editing 100

Gene Editing Clinical Trials Clinical Trials Genetics 100

Outsourcing Pharma

JULY 23, 2020

A leader from the genetic services company discusses how use of telemedicine can help pull in patients and keep trials going.

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Genetics Trials Clinical Development Development 52

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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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. Merck will also make royalty payments on any approved products developed out of the partnership.

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Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Although a high number of cell and gene therapies are in early-stage development, a new wave of approvals could be on its way. Securing the supply chain.

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Gene Therapy Gene Medicine Genetics 245

Scienmag

JUNE 8, 2021

Data supports QurAlis’ approach to treat hyperexcitability induced neurodegeneration in ALS patients; QurAlis gears up for clinical development of therapeutic candidate, QRL-101 CAMBRIDGE, Mass.–(BUSINESS

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The Pharma Data

DECEMBER 13, 2020

As part of the collaboration, Hummingbird will be leveraging Tempus’ AI-enabled platform and proprietary data, as well as joining its TIME Trial® Network, for rapid identification, site activation and efficient enrollment of cancer patients who have NRG1 fusions and meet eligibility criteria for HMBD-001 clinical trials.

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Pharmaceutical Technology

MARCH 4, 2023

Treprostinil SR is under clinical development by Camurus and currently in Phase II for Raynauds Disease. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. It acts by targeting prostacyclin receptor.

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Drugs Development Clinical Trials Clinical Trials 100

Pharmaceutical Technology

MARCH 4, 2023

Treprostinil SR is under clinical development by Camurus and currently in Phase II for Raynauds Disease. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. It acts by targeting prostacyclin receptor.

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Drugs Development Clinical Trials Clinical Trials 100

Scienmag

FEBRUARY 10, 2021

today announced a strategic research and development collaboration to expand the evaluation of Mirati’s two investigational small molecule, potent and selective KRAS inhibitors – adagrasib (MRTX849), a G12C inhibitor in clinical development, and MRTX1133, a G12D inhibitor in […].

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