Drug Target Review

SEPTEMBER 10, 2024

For example, tox material from a pool of clones will contain a potentially broader profile of product quality attributes (eg glycan profile, high molecular weight species, etc) in addition to potentially slightly higher relative amounts of process impurities which can resemble the worst-case scenario during the toxicology testing.

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The Pharma Data

MAY 2, 2022

The target of DF7001 is 5T4, a protein expressed on cancer cells and stromal cells that support tumor growth associated with poor prognosis in several cancers, including non-small cell lung cancer (NSCLC), pancreatic cancer, breast cancer, and head and neck squamous cell carcinomas (HNSCC). View the full release here: [link].

Licensing 52

Licensing Licensing Research Protein Expression 52

The Pharma Data

DECEMBER 29, 2020

NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,

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The Pharma Data

AUGUST 16, 2021

The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.

Immune Response 52

Immune Response Vaccine Virus RNA 52

Drug Target Review

MAY 4, 2023

A cure for sickle cell anaemia A recent scientific breakthrough in the treatment of sickle cell anaemia —a genetic disorder marked by episodes of intense pain—illustrates how combining GWAS with cutting-edge molecular tools like gene editing can identify causal variants and lead to innovative therapies.

Disease 52

Disease Small Molecule Protein Expression Research 52

The Pharma Data

APRIL 8, 2021

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We “Following U.S.

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Clinical Trials Trials Disease Therapies 52

The Pharma Data

OCTOBER 28, 2020

In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40

The Pharma Data

APRIL 26, 2022

This investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi, the bacteria that cause Lyme disease. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick.

Vaccine 52

Vaccine Disease Protein Expression Trials 52

The Pharma Data

SEPTEMBER 10, 2020

Basel, 11 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data that show OCREVUS® (ocrelizumab) is a highly effective treatment option for people with relapsing-remitting multiple sclerosis (RRMS) who experienced a suboptimal response to their prior disease modifying therapy (DMT). and primary progressive MS (PPMS).

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Treatment Disease FDA Approval Clinical Trials 52

The Pharma Data

SEPTEMBER 2, 2020

Roche’s Chief Medical Officer and Head of Global Product Development. From the success of our first-in-class B-cell MS therapy OCREVUS, we are poised to continue advancing the science in MS with our new investigational BTK inhibitor fenebrutinib, and in NMOSD with the recent FDA approval of ENSPRYNG.”.

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Clinical Trials Treatment Disease Therapies 52

The Pharma Data

SEPTEMBER 8, 2020

Roche’s Chief Medical Officer and Head of Global Product Development. “In In addition, over 170,000 people have been treated with OCREVUS, our first-in-class B-cell therapy, and we are incorporating years of clinical trial data and real-world evidence to optimise its potential to improve outcomes for patients with MS.”.

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Clinical Trials Trials Disease FDA Approval 52

Codon

APRIL 14, 2025

Leveraging transparency to drive competition has worked well in other fields, such as consumer products , economic policy , and cloud services. One such opportunity, for example, is in protein expression. Suddenly, operational excellence could become a competitive advantage and straightforward to evaluate.

Protein Expression 84

Protein Expression Engineering Research RNA 84

Drug Target Review

MAY 22, 2024

Secondly, circRNAs can be engineered for more efficient protein expression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. There are three major reasons for why Circio has selected AATD as its lead gene therapy program.

Therapies 59

Therapies RNA Protein Expression DNA 59

Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

Vaccine 52

Vaccine Therapies Clinical Trials Small Molecule 52

Drug Target Review

MARCH 15, 2024

Advancements in cell line development (CLD), including screening and selection of final clones, scale up, automation and digitalisation have enabled precision and enhanced productivity. Efforts have focused on Chinese hamster ovary (CHO) cell line improvements since it is a commonly used host for the expression of therapeutic proteins.

Engineering 105

Engineering Molecular Biology Protein Expression Medical Schools 105

The Pharma Data

JANUARY 16, 2021

Etta Biotech”), to set up a high titer transient protein expression platform for high quality protein production using JS Bio’s transient transfection media. JS Bio becomes the exclusive cell culture supplier for Etta Biotech’s transient transfection high titer protein expression platform.

Protein Expression 52

Protein Expression Protein Production Production Drug Development 52

The Pharma Data

DECEMBER 3, 2020

An initial analysis of data from 17/22 patients who completed one of these assessments showed substantially decreased neurocognitive abilities compared to children of the same age level despite the use of multiple anti-epileptic therapies. .–( BUSINESS WIRE )– Stoke Therapeutics , Inc. STK-001 is designed to upregulate Na V 1.1

Clinical Trials 52

Clinical Trials Protein Expression Pharmacokinetics Trials 52

Drug Target Review

SEPTEMBER 4, 2023

Studies have shown that targeting some expression gene sites could lead to the production of new drugs as indicated. The basic role of sirtuins is that they remove acetyl groups from other proteins and they could be a potential drug target.

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Drugs RNA DNA Disease 111

The Pharma Data

SEPTEMBER 28, 2021

The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to 6 weeks aged and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne dystrophy (DMD).

Clinical Trials 52

Clinical Trials Protein Expression Therapies FDA 52

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.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. AI + Bio *Transfer learning for cross-context prediction of protein expression from 5’UTR sequence. Molecular Therapy. Gene Therapy. Subramanian M.

Therapies 52

Therapies RNA Engineering Vaccine 52

The Pharma Data

MARCH 26, 2021

Roche’s Chief Medical Officer and Head of Global Product Development. “We Tecentriq also has four approved indications in NSCLC as either a single agent or in combination with targeted therapies and/or chemotherapies. If approved, Tecentriq would provide a new treatment option, free from chemotherapy-related adverse effects.

Treatment 52

Treatment Therapies Protein Expression Immune Response 52

The Pharma Data

JULY 21, 2021

This investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi , the bacteria that cause Lyme disease. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

The Pharma Data

MAY 4, 2021

Roche’s Chief Medical Officer and Head of Global Product Development. Tecentriq monotherapy has been shown to improve overall survival in people with high PD-L1 expression, when compared to chemotherapy, and therefore represents a new treatment option for people living with this difficult-to-treat disease.”.

Treatment 52

Treatment Therapies Disease Pharmaceuticals 52

The Pharma Data

JANUARY 13, 2021

Germany-based Cevec Pharmaceuticals GmbH signed a licensing agreement with Biogen for the use of its proprietary ELEVECTA Technology for the manufacturing of adeno-associated virus (AAV) vectors for gene therapy applications. The deal will provide Biogen the rights to use the technology across their portfolio of gene therapy products.

Licensing 52

Licensing Licensing Clinical Trials Vaccine 52

The Pharma Data

JANUARY 13, 2021

GMP Production of TNX-1500 is Expected to be Available in the Third Quarter of 2021. Dr. Lederman continued, “Despite the recognized promise of anti-CD40-ligand mAb therapy, first generation anti-CD40-ligand mAbs were limited because their crystallizable fragment (Fc) domain interacted with a cell surface receptor called Fc?RII,

Pharmaceuticals 52

Pharmaceuticals Small Molecule Vaccine Protein Expression 52

Conversations in Drug Development Trends

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. Other researchers are investigating approaches that use AAV-mediated trophic support and antibody-based therapies, such as vaccines and small molecule drugs, to modify various protein expressions.

Treatment 81

Treatment Clinical Trials Small Molecule Therapies 81

The Pharma Data

OCTOBER 26, 2020

Responses were observed in all primary tumor types and across all lines of prior therapy. Responses were observed in all primary tumor types and across all lines of prior therapy. This response developed as early as D43 and sustained at 6 months after initiation of therapy, consistent with the durable disease-control.

Vaccine 40

Vaccine Trials Treatment Virus 40

FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance states that only one 90-minute pre-BLA meeting will typically be granted for a specific product or indication planned for an original marketing application.

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Therapies FDA Production Disease 59