DrugBank

JULY 24, 2024

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

Small Molecule 98

Small Molecule Drugs Clinical Trials Therapies 98

LifeSciVC

JUNE 12, 2025

This concept of effecting a protein change. was based on experience building on the understanding that a single protein variant, could have markedly different outcomes for patients with chronic kidney disease. We built our proprietary OPERA platform ( here ), leveraging oligonucleotide guided RNA editing to modulate protein expression.

RNA 67

RNA Protein Expression DNA Science 67

The Pharma Data

MARCH 30, 2021

Broad expression of FAP demonstrated in tumors or in tumor stroma across many solid tumors 1 ,2 ,3. Novartis Oncology continues to reimagine cancer care through development of robust radioligand therapy portfolio. We continue to invest in radioligand therapy as one of the four unique platforms of Novartis Oncology.

Therapies 52

Therapies Licensing Licensing Protein Expression 52

Conversations in Drug Development Trends

AUGUST 14, 2024

Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness. Fortunately, personalized treatment plans help circumvent therapy resistance while optimizing response rate and improving quality of life.

Treatment 78

Treatment Therapies Disease Protein Expression 78

Drug Target Review

FEBRUARY 19, 2024

A pivotal moment for me was when I realised that if I understood disease mechanisms on a molecular level, I could be part of an industry that helps develop medicines/therapies for those who need them most. I loved learning how to explain at the molecular level what was happening in nature, in human beings and disease settings.

Molecular Biology 110

Molecular Biology Science Protein Expression Production 110

PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type.

Laboratories 52

Laboratories Clinical Trials Protein Expression Pharmacokinetics 52

The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021.

RNA 52

RNA Therapies Disease Protein Expression 52

Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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Drug Development Treatment Drugs FDA Approval 52

The Pharma Data

NOVEMBER 26, 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.,

Disease 52

Disease Treatment Clinical Trials FDA 52

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

AUGUST 16, 2021

The vaccine candidate, presently at a preclinical development stage, is a non-chemically modified mRNA, encoding the prefusion stabilized full-length spike protein of the SARS-CoV-2 virus, and formulated within Lipid Nanoparticles (LNPs). CureVac had its initial public offering on the New York Nasdaq in August 2020.

Immune Response 52

Immune Response Vaccine Virus RNA 52

The Pharma Data

JUNE 7, 2023

3,4,5 TROP2 is a protein expressed in more than 90% of NSCLC tumors. Rahway, NJ, USA), an anti-PD-1 therapy. 3,4,5 TROP2 is a protein expressed in more than 90% of NSCLC tumors. Rahway, NJ, USA), an anti-PD-1 therapy. More than one million people are diagnosed with NSCLC at an advanced stage each year.

Trials 40

Trials Protein Expression Therapies Treatment 40

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., Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development. Claims Database.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 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).

Treatment 52

Treatment Disease FDA Approval Clinical Trials 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.,

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

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 2, 2020

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.”. Roche’s Chief Medical Officer and Head of Global Product Development.

Clinical Trials 52

Clinical Trials Treatment 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

Drug Target Review

SEPTEMBER 10, 2024

For example, advances in media and feeds have contributed to higher cell protein expression by optimising nutrient availability and reducing accumulation of waste products within a culture. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.

Drug Development 65

Drug Development Clinical Supply Production Protein Expression 65

The Pharma Data

SEPTEMBER 8, 2020

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.”. Roche’s Chief Medical Officer and Head of Global Product Development. “In

Clinical Trials 52

Clinical Trials Trials Disease FDA Approval 52

The Anticancer Fund

SEPTEMBER 29, 2021

Effect of Celecoxib vs Placebo as Adjuvant Therapy on Disease-Free Survival Among Patients With Breast Cancer: The REACT Randomized Clinical Trial Published in JAMA Oncology This large (n=2639) trial of adjuvant celecoxib vs. placebo in women with HER2-negative primary breast cancer finds no effect on disease free survival at 10-years follow up.

Clinical Trials 40

Clinical Trials Drugs Trials Protein Expression 40

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. “As

Disease 142

Disease Therapies Clinical Trials Engineering 142

Codon

APRIL 14, 2025

One such opportunity, for example, is in protein expression. When working with proteins — or designing new ones — scientists must first express them within a cellular “host,” which in turn demands that the proteins be successfully translated and folded into a stable, soluble structure.

Protein Expression 91

Protein Expression Engineering Research RNA 91

ASPET

JUNE 27, 2024

Estrogen receptor (ER)-negative breast cancers are known to be aggressive and unresponsive to anti-estrogen therapy, and triple negative breast cancers are associated with poor prognosis and metastasis. Thus, new targeted therapies are needed.

Therapies 100

Therapies Protein Expression 100

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

Chemical Biology and Drug Design

MARCH 5, 2024

Curcumin modulates the gene and protein expression levels of ferroptosis mediators via JNK signaling. Ferroptosis, a new form of regulated cell death, plays a vital role in the pathogenesis and therapy of cancers.

Protein Expression 100

Protein Expression Regulations Therapies Treatment 100

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

The Pharma Data

APRIL 27, 2022

Based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a significant improvement in both progression-free survival and overall survival Enhertu has now been granted five Breakthrough Therapy Designations, including three in breast cancer and one in both lung and gastric cancers.

Therapies 52

Therapies Trials Protein Expression Treatment 52

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. About JS Bio.

Protein Expression 52

Protein Expression Protein Production Production Drug Development 52

Conversations in Drug Development Trends

APRIL 7, 2025

This approach improves treatment outcomes over traditional broad-spectrum therapies, which are prone to more side effects and adverse events. Carefully crafted enrichment strategies optimize recruitment and enrollment, ensuring that the included participants will likely respond to the therapy.

Research 40

Research Clinical Trials Trials Clinical Research 40

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

MARCH 15, 2024

As discussed above, recent advances in gene editing tools and transposon-based vectors have facilitated targeted and consistent cell line engineering for the development of protein expressing CHO cell lines. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014. Biotechnol Prog. 2017 Nov;33(6):1468-1475.

Engineering 105

Engineering Molecular Biology Protein Expression Medical Schools 105

Drug Target Review

SEPTEMBER 4, 2023

10 Reported cAMP as the second messenger signalling molecule conserved from bacteria to humans and modulates several biological processes, including protein expression, gene transcription, and cell development and differentiation.

Drugs 111

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

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. McGrady N.R.

Therapies 52

Therapies RNA Engineering Vaccine 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