Pharmaceutical Technology

NOVEMBER 8, 2022

Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.

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Gene Therapy Gene Development Genetics 305

FDA Law Blog

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Pharmaceutical Technology

MARCH 24, 2023

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.

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AuroBlog - Aurous Healthcare Clinical Trials blog

JUNE 8, 2023

The artificial sweetener sucralose (marketed as Splenda) is widely used and found in products like diet soda and chewing gum. As DNA holds the genetic code controlling how our bodies grow and are maintained, that’s a serious problem that […]

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DNA Scientist Genetics Production 245

Pharma Mirror

APRIL 28, 2021

The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine. Catalent will further support process optimization and look to reduce material.

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Pharmaceutical Technology

MAY 25, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) aims to launch a pilot genetic biobank that will gather patient data to associate drug-related adverse events to their genetic makeup. The first patients will have their genetic makeup sequenced in Spring 2024 and this data will be shared in 2025.

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Biobanking Genetics Genome Project Genome 130

Pharma Times

MAY 2, 2024

The inherited disorder is caused by genetic mutations that affects haemoglobin production

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Pharma Times

APRIL 14, 2023

Product delivers epigenetic information from a single low-input DNA library without medical treatment

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Genetics DNA Production 123

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia thalassemia in adult and paediatric patients.

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Gene Therapy Gene Genotype In-Vivo 246

Pharmaceutical Technology

OCTOBER 19, 2022

Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 AK-OTOF is the lead product candidate of the company. for each share in cash. The deal comprises an acquisition value of $12.50

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Pharmaceutical Technology

JANUARY 16, 2023

The fully human, autologous BCMA CAR T-cell product candidate of CARsgen, CT053 has been developed to treat relapsed/refractory multiple myeloma (R/R MM). It comprises autologous T cells that are modified genetically with a CAR including a complete human anti-BCMA single-chain fragment variant that has a high binding affinity.

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Medicine Gene Therapy Production Gene 298

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. According to GlobalData, Phase I drugs for Brain Tumor does not have sufficient historical data to build an indication benchmark PTSR for Phase I.

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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. According to GlobalData, Phase I drugs for Brain Tumor does not have sufficient historical data to build an indication benchmark PTSR for Phase I.

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Genetic Engineering Engineer Genetics Clinical Trials 100

Pharmaceutical Technology

SEPTEMBER 5, 2022

The National Medical Products Administration of China (NMPA) has approved CanSino Biologics ’ (CanSinoBIO) recombinant Covid-19 vaccine (Adenovirus Type 5 Vector) for inhalation, Convidecia Air, as a booster. This vaccine leverages the same adenovirus vector technological platform as Convidecia, the intramuscular version.

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Pharmaceutical Technology

JUNE 29, 2022

For hundreds of years, we have found myriad uses for microbial enzymes in manufacturing – from food, drink, and household products to a range of industrial applications. In 2021, Novo Nordisk Pharmatech, a leading pharmaceutical-grade insulin and Quats product supplier announced plans to enter the enzyme market.

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STAT News

APRIL 17, 2023

Genetic engineering has the potential to transform how we raise animals for meat and other products, making food safer, improving animal health and welfare, and shrinking animal agriculture’s environmental footprint. Pigs that are less likely to induce allergic reactions in humans.

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Genome Genomics Genetic Engineering Production 98

World of DTC Marketing

DECEMBER 29, 2020

First, a novel approach was used that didn’t require traditional vaccine production in cell cultures or eggs. These vaccines were developed using different “platform technologies” that involve slotting genetic material from the virus into a tried and tested delivery package. 1455NO-HEALTH-CORONAVIRUS_VACCINES_PFIZER_O_.

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Medical Xpress

JANUARY 25, 2023

A new study from researchers at Laval University in Quebec, Canada, shows that genetically increasing omega-3 fatty acid production, without direct gut microbiota contact, improves the balance of insulin and glucagon (glucose balance) in obese mice.

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Pharmaceutical Technology

NOVEMBER 7, 2022

Leveraging its cell expansion and activation technology as well as cell manufacturing capabilities, the company can extend NK cells while substantially boosting cytotoxicity across peripheral blood-derived products.

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Gene Therapy Clinical Trials Clinical Trials Trials 246

Pharmaceutical Technology

MAY 17, 2023

They will also have the potential to treat diseases that are caused by too little protein by increasing production, thus restoring the protein to normal levels. Antisense therapies are designed for destroying mRNA, reducing the amount of disease-causing protein.

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Pharmaceutical Technology

DECEMBER 12, 2022

An investigational late-stage product candidate, CART-ddBCMA is being analysed in the Phase II iMMagine-1 clinical trial at present. It leverages the new D-Domain binder of Arcellx and contains autologous T cells genetically modified for targeting multiple myeloma.

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Pharmaceutical Technology

FEBRUARY 7, 2023

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for Takeda ’s dengue virus vaccine candidate, Qdenga (Dengue Tetravalent Vaccine [Live, Attenuated]). The vaccine candidate has been approved for active immunisation against the infection in people from four years of age.

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Pharmaceutical Technology

JUNE 24, 2022

The protein-based vaccine is engineered from the genetic sequence of the SARS-CoV-2 virus’ initial strain. Earlier this month, the US FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 21 to 0 recommending the EUA grant for Novavax’s NVX-CoV2373 for individuals aged 18 years and above.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. What Does the Future of Gene Editing Hold?

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XTalks

DECEMBER 4, 2024

Conventional resveratrol products often face challenges like poor absorption and significant gastrointestinal side effects. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.

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Bioavailability Genetic Disease Marketing RNA 111

Pharmaceutical Technology

MAY 24, 2023

Streamlining the regulatory process, the AMP BGTC will also facilitate cost-efficient vector production to increase access for patients with rare and ultra-rare genetic diseases. The collaboration creates a reusable standardised method that minimises upfront costs and helps to reduce development barriers.

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Gene Therapy Gene Development Manufacturing 242

Pharmaceutical Technology

JULY 6, 2022

A protein-based vaccine, NVX-CoV2373 is made from the genetic sequence of the SARS-CoV-2 virus’ first strain. The latest development comes after the Committee for Medicinal Products for Human Use of the European Medicines Agency granted a positive recommendation in June this year.

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FDA Law Blog

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Pharmaceutical Technology

JUNE 29, 2022

A CD19-directed genetically modified autologous T cell immunotherapy, Yescarta is indicated for FL patients who have received three or more previous lines of systemic therapy. The treatment has maintained orphan medicinal product designation for this indication.

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Gene Therapy Clinical Trials Clinical Trials Genetics 278

XTalks

MARCH 17, 2025

Recent pre-mass production trials tests conducted to ensure that the devices meet performance and manufacturing quality standards before full-scale production demonstrated that the A+Pre chip recovered 94% of tumor cells, while the AC-1000 achieved a 79.5% recovery rate, surpassing their performance targets.

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Pharmaceutical Technology

FEBRUARY 9, 2023

Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications. The company is focused on developing RNA-targeting small molecule therapies to treat cancer and other serious genetically defined diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MAY 24, 2023

The ability to modify or introduce genetic material in human cells in such a precise and patient-centered manner clearly constitutes a breakthrough in personalized medicine. Allogeneic therapies begin with healthy donor samples to develop the eventual therapeutic product which can be administered to multiple patients.

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Pharmaceutical Technology

DECEMBER 8, 2022

Developing neoantigen products needed for the therapy has faced difficulties including lead time to get GMP DNA and complicated supply chains. With a five-day manufacturing process, the enzymatic production approach of Touchlight will back Odimma in developing its programme. . It is manufactured using an enzymatic production process.

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DNA Genetics Manufacturing Production 147

BioPharma Reporter

JANUARY 2, 2023

Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.

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Gene Therapy Genetics Gene Production 118

Medical Xpress

NOVEMBER 8, 2022

Researchers at the University Hospital Bonn (UKB) and the Technical University (TU) Dresden have investigated an underlying mechanism and deciphered the driving force of uncontrolled interferon production through studying the rare autoimmune disease Aicardi-Goutières syndrome.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

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Gene Therapy Gene Gene Editing Development 243

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. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine.

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