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

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. Could you give us an overview of the Lineage platform?

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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 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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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.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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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

To target proteins involved in causing or spreading disease, typically a drug will obstruct a protein’s active site so it cannot function. Proteins can become part of a cancerous tumour and allow for its unrestricted growth, cause diseases like Alzheimer’s , and build up in the heart to impact how it pumps blood around the body.

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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

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Miraculously, there was an approved treatment. This treatment only exists because a small charity in the ’70s donated the funds needed for the researcher to discover it. Uplifting Athletes (UA) is on a mission to change that. It saved my life.”

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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

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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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 . Varricchi G, et al. Li Y, et al.

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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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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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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

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”. About ERLEADA ® (apalutamide).

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The Pharma Data

MAY 5, 2022

Validated Measurable Residual Disease Assays are Critical for the Rapid Development of Future Novel Therapeutics for Patients with Acute Myeloid Leukemia. 1 Clinical trials for new therapies in AML are becoming increasingly lengthy to complete based on the traditional clinical endpoint of survival. About Acute Myeloid Leukemia.

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Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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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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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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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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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

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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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

SEPTEMBER 18, 2020

Based on this recommendation, a final decision regarding approval of Tecentriq in combination with Avastin in this disease setting, along with the full details of the approved indication, is expected from the European Commission in the near future. 0.76; p<0.0001), compared with sorafenib.

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Drug Discovery World

APRIL 25, 2023

Spin-out companies play a vital role in translating Cancer Research UK-funded research from the lab into life-saving treatments. Alba Health which is on a mission to reduce chronic disease risks from childhood, leveraging data science and the latest microbiome science. billion by 2030.

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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 If the body fails to make the right protein, diseases such as cancer, type II diabetes, neurological conditions, and metabolic diseases can occur.

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Drug Discovery World

OCTOBER 6, 2022

This growth is due to factors including a rise in speciality medicines, an increase in lifestyle-driven diseases and a growing population 1. France, Switzerland, and the United Kingdom have seen the fastest growth, accounting for 63% of the biotechs founded between 2018 and 2020.” Valued at $14.8 until 2030. Hotspots . References: .

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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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Agency IQ

MARCH 29, 2024

FDA’s oncology advisory committee to consider MRD as a multiple myeloma endpoint Yesterday, the FDA announced that its Oncologic Drugs Advisory Committee would weigh in on the potential use of minimal residual disease (MRD) as an endpoint in multiple myeloma clinical trials. immune cells and other blood cell types) lack.

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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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Drug Discovery World

MARCH 29, 2023

UMNs have been defined in the context of the draft as diseases where there’s a lack of good treatments, and which have a high burden 3. UMNs have been defined in the context of the draft as diseases where there’s a lack of good treatments, and which have a high burden 3.

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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 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

MARCH 16, 2021

Viela (Nasdaq: VIE), headquartered in Gaithersburg, Maryland, is a biotechnology company dedicated to the discovery, development and commercialisation of novel treatments for autoimmune and severe inflammatory diseases. AstraZeneca.

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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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