Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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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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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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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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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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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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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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New Drug Approvals

APRIL 2, 2025

1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA. 11] The FDA granted the application for suzetrigine priority review , fast track , and breakthrough therapy designations. [2]

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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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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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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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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 6, 2021

Approvals were based on the overall response rate (ORR) from the open-label, single-arm Phase II CHRONOS-1 (NCT01660451) trial of copanlisib monotherapy in 104 adult patients with follicular B-cell NHL who had relapsed disease following at least two prior systemic therapies. EU, UK and Japan. Filings in other regions are underway or planned.

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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

JULY 18, 2021

The emergency use of this product is only authorized for the duration of the declaration that circumstances exist justifying the authorization of emergency use of the medical product under Section 564 (b) (1) of the FD&C Act unless the declaration is terminated or authorization revoked sooner.

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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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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

OCTOBER 6, 2021

Pfizer and BioNTech today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion on the administration of the companies’ COVID-19 vaccine as a booster dose at least six months after the second dose in individuals 18?years years of age and older.

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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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Alta Sciences

AUGUST 29, 2024

Listen or read Issue 33 of The Altascientist on altasciences.com , or wherever you listen to podcasts: GROWING NEEDS FOR CNS THERAPY SOLUTIONS The different parts of the nervous system, including the brain and spinal cord and the peripheral nervous system, are important drug targets for many serious diseases affecting human health.

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The Pharma Data

JANUARY 19, 2021

TCR 2 is developing novel T-cell therapies for solid tumors and hematological cancers. Other products include SY-2101, a novel oral form of arsenic trioxide for acute promyelocytic leukemia, and SY-5609, a highly selective and potent oral CDK7 inhibitor for solid tumors. The company is based in Suzhou and Shanghai, China.

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The Pharma Data

DECEMBER 17, 2020

He previously served as vice president, for drug device industrialization at AbbVie, where he led product scale-up and industrialization projects for drug-device combinations. Goater is CEO at Surface Oncology, an immuno-oncology company developing next-generation antibody therapies. Previously he was CFO of Voyager Therapeutics.

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The Pharma Data

JANUARY 10, 2021

Chiauranib as a single agent therapy inhibits the overactive Aurora B pathway existed in and related to molecular mechanism of SCLC, which is an important feature of Chiauranib distinct to other antiangiogenic drugs. SH ) was founded in Shenzhen on 2001, specializing in the research and development of novel small molecule drugs.

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The Pharma Data

MARCH 23, 2022

“These positive results demonstrate that etrasimod, if approved, could be a potential breakthrough option for patients with ulcerative colitis who aren’t able to experience improvement on current therapies,” said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development.

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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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The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) for the initiation of a Phase 1 clinical trial to evaluate IDE397, a small molecule methionine adenosyltransferase 2a (MAT2A) inhibitor, for the treatment of patients having solid tumors with methylthioadenosine phosphorylase (MTAP) deletion. Chief Scientific Officer, IDEAYA Biosciences.

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The Pharma Data

SEPTEMBER 25, 2020

Monte Rosa Therapeutics has raised $96m in Series B financing to support further develop of its pipeline of small-molecule protein degraders. With this financing, we are now well positioned to broadly develop our integrated drug discovery platform and advance multiple new therapies toward clinical development,” he added.

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