PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

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Drug Target Review

OCTOBER 11, 2023

Most of these conditions are genetic in origin and the majority have no effective treatment. 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.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

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Drug Discovery World

DECEMBER 18, 2023

Casgevy The committee recommended granting a conditional marketing authorisation for Casgevy (exagamglogene autotemcel), an advanced therapy medicinal product (ATMP) for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease.

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Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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Drug Target Review

NOVEMBER 7, 2023

Chemists at Stanford University discovered how one of the pathways leading to this protein “woodchipper” works furthering the understanding of new therapeutics for autoimmune diseases, age-related disorders, and treatment-resistant cancers. Enzyme replacement therapy is a frequent treatment for these disorders.

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Common Sense for Drug Policy Blog

JUNE 4, 2023

Opioid Substitution Treatment in the UK "Opioid substitution therapy (OST) is an evidence-based treatment for opioid dependency, with treatment engagement shown to be protective against drug related deaths ( Santo et al., 2019 ; Public Health England, 2018 ). dwf_admin Sun, 06/04/2023 - 20:03 Source Poulter, H.

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Drug Discovery World

JUNE 14, 2023

The recent clinical trial for metastatic breast cancer patients (DESTINY-Breast04) found that a treatment traditionally used for HER2 positive patients was also effective in patients considered HER2 negative. It used the current CAP/ASCO testing algorithm for HER2 protein expression levels.

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Drug Discovery World

SEPTEMBER 1, 2023

The new therapeutics companies BII has accepted into the Venture Lab acceleration programme are: Loma Therapeutics, which is developing targeted immunotherapy for efficient and safe treatment of patients with HPV infection, dysplasia, and cancer. Since its inception in 2018, BII has supported 87 start-ups and projects.

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The Pharma Data

DECEMBER 17, 2020

18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.

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Drug Target Review

JANUARY 25, 2024

These findings offer a novel strategy for developing targeted treatments for triple-negative breast cancer, which currently has no approved options. The peptide in development is based on a 2018 Cleveland Clinic discovery and serves as a proof-of-concept for this type of drug for triple-negative breast cancer. Available from: [link]

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The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. 22, 2020 — Tagrisso ( osimertinib ) received approval as an adjuvant treatment for patients with non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, the U.S. Professional. TUESDAY, Dec.

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Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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Drug Discovery World

JULY 22, 2022

Although immunotherapy has revolutionised cancer therapy, the effectiveness of currently available immunotherapies varies significantly across patients and treatment types. Immune checkpoint inhibitors (ICIs) like pembrolizumab (Keytruda) have numerous benefits and have become a standard of treatment for many patients.

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Drug Discovery World

JUNE 1, 2023

According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1. A market research report published in late June 2018 by HTF market Intelligence Consulting suggested that the ADC market in China would grow exponentially by 2023 4.

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The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. In addition, health-related quality of life (HRQoL), per total Functional Assessment of Cancer Therapy–Prostate (FACT-P), continued to be maintained with ERLEADA ®.

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Drug Discovery World

OCTOBER 4, 2022

Oligomerix’s portfolio of compounds is expected to provide a potentially lower-cost treatment alternative and/or complement to the newly emerging high-cost therapeutic options such as the monoclonal antibody products. Global dementia prevalence is expected to increase to 152 million in 2050, a 204% increase compared to 2018.

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The Pharma Data

APRIL 17, 2021

TECFIDERA was approved under the National Medical Products Administration priority review process evaluating therapies with urgent clinical needs. Biogen’s expansion in China now includes treatment options approved for relapsing MS and spinal muscular atrophy. We thank the NMPA for undertaking priority review to approve TECFIDERA. “We

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The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment. Over-the-counter treatments fail due to the body’s natural protective system. 25% for dry eye disease treatment.

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Drug Discovery World

SEPTEMBER 26, 2022

AstraZeneca’s Tezspire (tezepelumab) has been approved in the European Union as an add-on maintenance treatment in patients 12 years and older with severe asthma who are inadequately controlled with high dose inhaled corticosteroids plus another medicinal product. . 2018; 9: 1595. Official comments . N Engl J Med. 2017;377: 936-946.

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Common Sense for Drug Policy Blog

JANUARY 5, 2024

of the US adult population, injected drugs in 2018. Much of this increase is likely attributable to increases in IDU, but it is important to consider methodological differences in the creation of this 2018 estimate vs the 2011 estimate. million PWID in 2011, which suggest the 2018 estimate is closer to 3 times higher than in 2011.

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Drug Discovery World

AUGUST 10, 2022

Until about 25 years ago, the best treatment option for someone with blood cancer was either toxic radiation and chemotherapy or a bone marrow transplant that posed additional life-threatening risks to the patient 1. Next, the advent of antibody treatments in 1997 created the first targeted cancer therapy 2.

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The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

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The Pharma Data

NOVEMBER 24, 2020

25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).

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Agency IQ

OCTOBER 6, 2023

In a first, FDA tackles treatments for stimulant use disorder A new draft guidance from the FDA gives developers a roadmap to advance the development of novel therapies to address stimulant use disorders. SAMSHA published an “ evidence-based resource guide ” on substance use disorder treatment in June 2020.

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The Pharma Data

JUNE 29, 2021

ENSPRYNG is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training. Importantly, people with NMOSD now have the flexibility to administer treatment at home, which may alleviate the need to travel for hospital appointments.”.

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The Pharma Data

AUGUST 19, 2021

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].

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Drug Target Review

AUGUST 24, 2023

We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.

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Drug Target Review

SEPTEMBER 4, 2023

7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria. 2018): Ubiquitin Proteasome pathway proteins as potential drug targets in parasite Trypanosoma cruzi, Scientific Reports. (8):8399. link] Ojewale Kayode (2018).

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Drug Discovery World

OCTOBER 6, 2022

France, Switzerland, and the United Kingdom have seen the fastest growth, accounting for 63% of the biotechs founded between 2018 and 2020.” While half of that went into BioNTech and CureVac, companies working on non- Covid-19 related treatments also got important financing rounds.” . Hotspots . billion, equivalent to $3.7

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 We can see this versatility in the various treatments that are currently being researched.

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The Pharma Data

SEPTEMBER 18, 2020

Tecentriq in combination with Avastin was also recently included as a class I, A recommendation by the European Society for Medical Oncology (ESMO) for the treatment of unresectable HCC, as well as by many clinical practice guidelines globally. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

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The Pharma Data

MARCH 13, 2021

Takeda Pharmaceutical Company Limited ( TSE:4502/NYSE:TAK ) (“Takeda”) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

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The Pharma Data

SEPTEMBER 9, 2020

CERENOVUS Launches New Suite of Technologies to Advance Stroke Treatment. CERENOVUS Launches New Suite of Technologies to Advance Stroke Treatment. The announcement was made during the virtual European Society of Minimally Invasive Neurological Therapy (ESMINT). Stroke Research and Treatment, Hindawi, 27 Nov.

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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. The pre-IND program focuses on the treatment of prostate cancer, the second most commonly diagnosed cancer in men. Noria) and PSMA Therapeutics Inc.

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Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

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