Drug Target Review

DECEMBER 4, 2024

Confo Therapeutics , led by CEO Dr Cedric Ververken, is at the forefront of developing innovative GPCR-targeted therapies using its proprietary ConfoBody ® platform. In addition to generating agonistic antibodies, Confo is also successfully applying the ConfoBody/ConfoChimer technology to drugging GPCR with small molecules.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s? Join the club.

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Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

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Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise. Autophagy boosters, in contrast, are conventional small molecule drugs.

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Drug Target Review

OCTOBER 4, 2023

Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. 6 Combining the effect of payloads with different mechanisms of action – an approach that revolutionised small molecule chemotherapy – also holds the promise of enhanced therapeutic activity for ADCs.

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DrugBank

MAY 15, 2024

Researchers are experimenting with biologics—larger biological molecules that can do things small molecules can't, like targeting larger, more complex structures on cell surfaces or even inside cells. These methods could bypass the need to directly target these proteins with traditional drugs.

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. Biodistribution of the product (transgene/gene or cells) should be evaluated. How long does the study need to be?

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The Pharma Data

OCTOBER 15, 2020

Specifically, that entails improving manufacturing to control variables and thus ensure product consistency, and maturing the regulatory environment to improve predictability. Manufacturing cells is not like manufacturing small molecules,” Brian Culley, CEO of Lineage Cell Therapeutics , told BioSpace.

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Drug Target Review

JULY 23, 2024

This laid the groundwork for my career in drug discovery using novel modalities like small molecule inhibitors, antibodies, RNA interference (RNAi) and, currently, small molecule protein degraders – which are opportunities to change how we think about medicines.

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The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. In addition, Sosei Heptares will be eligible to receive tiered royalties on net sales of products resulting from the collaboration. TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D., About Sosei Heptares.

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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.

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The Premier Consulting Blog

NOVEMBER 20, 2024

It ensures that a production process consistently yields products of predetermined quality and safety. In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial small molecule or biologic product to expedite product availability for patients.

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The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

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Vial

DECEMBER 11, 2023

In the decades since its identification in 1989, advances in clinical research for new Hepatitis C therapies and the introduction of direct-acting antiviral agents (DAAs) have led to viral eradication in more than 98% of patients, resulting in HCV crossing from manageable to being the first curable , chronic viral infection. Zhao et al.

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Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

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The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS has been approved by Japanese regulatory authorities to manufacture Yescarta ® for the Japanese market, and commenced its supply of the product earlier this year. to Gilead Sciences K.K.,

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thought leadership

SEPTEMBER 29, 2023

Government begins its fiscal year on October 1, it signifies the annual revisions in FDA User Fees, which have an impact on applications and facilities associated with Prescription Drugs (including small molecules, vaccines, cell and gene therapy products, biologics, etc.), Generic Drugs, Biosimilars, and Medical Devices.

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The Pharma Data

JANUARY 25, 2021

–( BUSINESS WIRE )– Veralox Therapeutics, a biotechnology company developing first-in-class small molecule therapeutics that treat the underlying pathologies of diseases with significant unmet medical needs, today announced that the U.S. 26, 2021 13:00 UTC. FREDERICK, Md.–( About VLX-1005. Source link.

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New Drug Approvals

SEPTEMBER 23, 2024

The disease is caused by an expansion of uninterrupted trinucleotide CAG repeats in the “mutant” huntingtin (Htt) gene, leading to production of HTT (Htt protein) with an expanded poly-glutamine (polyQ) stretch, also known as a “CAG repeat” sequence. SCHEME PATENT PTC Therapeutics Inc.,

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DrugBank

AUGUST 15, 2024

To effectively navigate this ecosystem and expedite the development of new therapies, collaboration between the pharmaceutical industry and academia is proving increasingly vital. An example is the collaboration between Novartis and the University of Oxford to develop a gene therapy for spinal muscular atrophy, a rare genetic disease.

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The Pharma Data

SEPTEMBER 28, 2020

The product, known as INNA-051, is a synthetic small molecule therapy, self-administered once or twice weekly with an almost immediate effect.

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The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. With this acquisition, Bayer broadens its existing TAT development portfolio by adding an actinium-labeled PSMA-targeted alpha therapy.

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Drug Target Review

JULY 12, 2023

We also leverage existing clinical gene expression data from different subsets of molecularly profiled cancer patients, who differ in a certain aspect of disease or response to a particular therapy. We are pleased that to date our lead product candidate has aligned DCI profiles as expected.

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PPD

OCTOBER 18, 2023

There has been an unprecedented expansion of different types of pharmaceutical therapies, as well as a change in the overall approach to health care. Continued expansion of the biotech industry is occurring in parallel, with an estimated compound annual growth rate of 13.96% from 2023-2030.

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The Pharma Data

FEBRUARY 18, 2022

The company has introduced its approach to establishing scalable vaccine production by developing and delivering turnkey mRNA manufacturing facilities based on a container solution. This allows for mRNA vaccine production in bulk (mRNA manufacturing and formulation), while fill-and-finish will be taken over by local partners.

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The Pharma Data

JANUARY 13, 2021

Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small-molecule treatments for developmental and epileptic encephalopathies, other epilepsies, neuropathic pain, and tinnitus. REFERENCES.

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Trials Small Molecule Clinical Trials Treatment 52

The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. In particular, Editas is known to be exploring tunable CRISPR-Cas9 systems for therapies has developed self-inactivating Cas9 adeno-associated virus vectors.

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Dark Matter Blog

JULY 19, 2021

Or should I say back to retirement, as he was comfortably relaxing at home back in 2016 when Jen Petter lured him back into the fray with the siren call of developing a new class of RNA-targeted small-molecule medicines here at Arrakis. Here’s to many happy and productive years with Sue, Julia, Zooey, and all your guitars!

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The Pharma Data

APRIL 28, 2021

I am honored that Takeda receives two awards for the production and packaging of small molecule solid dosage form products,” said Thomas Wozniewski, global manufacturing & supply officer of Takeda. “In Takeda’s Hikari plant is located in the south of Japan in the Yamaguchi prefecture.

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The Pharma Data

NOVEMBER 15, 2020

As its name aptly suggests, Urovant is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for urologic conditions such as overactive bladder (OAB) and maladies involving adjacent areas of the human anatomy. . Urovant shareholders will receive $16.25 Source link.

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The Pharma Data

MAY 31, 2022

“More than 10 million people worldwide are affected by Parkinson’s disease, and there is a significant medical need for therapies to slow or stop the progression of this devastating neurodegenerative disease,” said Carole Ho, M.D., BIIB122 is an investigational small molecule inhibitor of LRRK2 that was discovered and developed by Denali.

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The Pharma Data

SEPTEMBER 10, 2021

– Abrocitinib is a once-daily oral JAK1 inhibitor indicated in Great Britain for the treatment of moderate to severe atopic dermatitis in patients aged 12 years and over, who are candidates for systemic therapy-. CIBINQO (abrocitinib) is an oral small molecule that selectively inhibits Janus kinase (JAK) 1.

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LifeSciVC

JANUARY 3, 2024

NF-kB is a key mediator of inflammatory responses, playing a crucial role in the regulation of cytokine production, as well as in the activation and proliferation of immune cells. One such pathway that we believe holds significant promise for a complex-directed therapeutic approach is the NF-kB pathway.

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The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. With the introduction of small-molecule medication, we open a new chapter in the fight against neurodegenerative diseases and have the chance to improve the lives of millions of patients drastically.”.

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Dark Matter Blog

JULY 16, 2021

My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I was enticed by Arrakis’ founder Jen Petter to serve in one more operational role, leading the research group at Arrakis as the company launched as one of the first biotechs to target RNA with drug-like small molecules.

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The Pharma Data

SEPTEMBER 8, 2021

Adds Rezurock™ (belumosudil) an FDA-approved, first-in-class treatment for adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. Sanofi has entered into a definitive merger agreement with Kadmon Holdings, Inc. Transaction Terms.

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