Pharmaceutical Technology

APRIL 28, 2023

Ocugen has received orphan drug designation from the US Food and Drug Administration for its OCU410ST (AAV5-hRORA) to treat ABCA4 – linked retinopathies. Utilising an AAV delivery platform, OCU410ST enables the delivery of the RAR-related orphan receptor A (RORA) gene to the retina.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Drug Discovery World

FEBRUARY 22, 2024

Following the first regulatory approval of a CRISPR-based drug in late 2023, over ten years since the CRISPR-Cas9 system was elucidated, there is considerable optimism about the future potential for gene editing technologies. appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 14, 2022

New research about how cancer-related proteins bind copper ions opens up potential new drug targets to treat cancer. . Higher levels of copper also mean more active copper-binding proteins. . Higher levels of copper also mean more active copper-binding proteins. . Memo1 and metastatic cancer. Image caption: ?The

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Drug Discovery World

JANUARY 25, 2023

The US Food and Drug Administration has also granted Orphan Drug designation for KB407 to treat patients with CF. The post Gene therapy for cystic fibrosis receives EC orphan drug designation appeared first on Drug Discovery World (DDW).

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Gene Therapy Gene Drugs Genetics 52

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. Using CRISPR Technology to Disrupt Networks of Immune Genes.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

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Gene Therapy Gene Protein FDA Approval 98

Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . In 2019, BV-101 was granted orphan drug designation in the European Union. . There are currently no approved disease modifying therapies for Huntington’s Disease.

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Pharmaceutical Technology

AUGUST 11, 2022

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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Drug Discovery World

JULY 24, 2023

The Defense Advanced Research Projects Agency (DARPA) has announced that Ginkgo Bioworks, which is building a platform for cell programming and biosecurity, has been awarded a four-year contract worth up to $18 million to reimagine how to manufacture complex therapeutic proteins.

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pharmaphorum

MARCH 19, 2021

Gain Therapeutics has priced its initial public offering, raising more than $40 million for its computational drug discovery platform and pipeline of drugs for diseases associated with protein misfolding. . The post Protein misfolding specialist Gain nets $40m from IPO appeared first on.

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Drug Discovery World

AUGUST 21, 2023

SBIR fast-track grant from the NIH to apply its proprietary Multiplexed Assays for the Rational Modulation Of Transcription Factors (MARMOT) platform to their deep learning AI model and accelerate the development of transcription factor drugs. Talus Bio was also awarded a $2.0M The post $4.3

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Drug Discovery World

FEBRUARY 13, 2024

It goes on to show that NX-2127, a potent targeted protein degrader with differentiated activity against BTK and IKZF1/3, can overcome this resistance across a broad range of acquired mutations. The post Protein degrader overcomes resistance in B-cell malignancies appeared first on Drug Discovery World (DDW).

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BioTech 365

OCTOBER 14, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Protein that manipulates gene expression inside the cell nucleus could inspire new RSV drugs, vaccines.Protein that manipulates gene expression inside the cell nucleus could inspire new RSV … Continue reading →

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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Pharmaceutical Technology

MARCH 27, 2023

Regenerative medicines in early-stage development (preclinical, discovery, or investigational new drug [IND]/ clinical trial application [CTA] filed status) have seen a change in drug targets compared to therapies in late-stage development (Phase II to pre-registration stage).

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pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Regardless of cohort, eligible participants are scheduled to receive the investigational gene therapy, either at the start of the study or after one year following treatment with placebo.

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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. The exact mechanism depends on the disease in question.

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Drug Discovery World

SEPTEMBER 6, 2023

The funding will support the development of the company’s gene therapy in a powder, a non-viral gene delivery system to improve tissue regeneration following surgery. The technology allows rapid transfection of patients’ cells with the gene(s) of interest.

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Scienmag

MAY 14, 2021

Approach opens a new avenue to study the biology of acute leukemia and for the development of new drugs Credit: Grembecka / Cierpicki Labs The protein made by the ASH1L gene plays a key role in the development of acute leukemia, along with other diseases.

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pharmaphorum

NOVEMBER 5, 2020

An experimental gene therapy developed by Texas biotech Genprex will be paired with AstraZeneca’s Tagrisso and Merck & Co’s Keytruda – both leading their respective drug classes in the treatment of non-small cell lung cancer (NSCLC).

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Pharmaceutical Technology

JANUARY 19, 2023

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

Drug Discovery World

APRIL 22, 2024

The discovery of two genes responsible for sensing the environment and adapting cell shape could offer potential new drug targets to stop cancer from metastasising. The researchers believe these genes, TIAM2 and FARP1, could be targeted to prevent melanoma cancer from metastasising.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Drug Discovery World

MARCH 22, 2024

This has been an interesting week for cell and gene therapies, with two landmark FDA approvals, two significant fundraising efforts and potentially ground-breaking study results in glioblastoma. The post This week in drug discovery (18-22 March) appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

JUNE 29, 2022

The pharmaceutical industry often relies on enzymes to perform a process known as biocatalysis, which facilitates the cost-effective and sustainable production of small molecule drugs at scale. The next wave of medicine is well on course to be cell and gene-based.

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Drug Discovery World

MAY 23, 2023

The US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) have voted eight to six in favour of a new gene therapy for Duchenne muscular dystrophy. SRP-9001 is intended to deliver a gene that codes for a shortened, functional form of dystrophin to muscle cells.

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XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . The authors have applied to the Food and Drug Administration for approval of the gel, which is called B-VEC. .

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Gene Therapy Gene Dermatology Clinical Trials 52

Drug Discovery World

MARCH 6, 2024

UK-based biotech SynaptixBio has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA). With the toxic protein suppressed, other proteins step in to help form normal myelin.

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Pharmaceutical Technology

AUGUST 11, 2022

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for Aro Biotherapeutics’ ABX1100 to treat Pompe disease. An investigational Centyrin-small interfering ribonucleic acid ( siRNA ) conjugate, ABX1100 acts on the glycogen synthase 1 (Gys1) gene in the muscle. ?Gys1

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Drug Discovery World

JANUARY 16, 2023

Vector BioPharma and the Instituto de Biologia Experimental e Tecnológica (iBET) have agreed a multi-phase collaboration to advance the technical development of Vector’s gene delivery platform. . Vector BioPharma’s gene delivery platform combines high-capacity virus-like particles with exogenous, high-avidity adapter proteins. .

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Drug Discovery World

MAY 29, 2023

The US Food and Drug Administration has approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a genetic disorder that affects the connective tissue in the skin and nails and results from mutation(s) in the COL7A1 gene.

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Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

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