The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. The purpose of the?Ph1

Trials 52

Trials Disease Clinical Trials Engineering 52

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.

Drug Development 52

Drug Development Disease Therapies Engineering 52

Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. 2019 Mar 1;55:93–100. 2019 Nov 1;8(6):784–8.

Drugs 111

Drugs FDA Disease Animal Testing 111

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

Disease 52

Disease Trials Treatment Clinical Trials 52

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.

Disease 52

Disease Clinical Trials Trials Treatment 52

The Pharma Data

OCTOBER 1, 2021

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. Deucravacitinib is being studied in multiple immune-mediated diseases, including psoriasis, rheumatoid arthritis , lupus and inflammatory bowel disease.

Disease 52

Disease Laboratories Clinical Pharmacology Treatment 52

The Pharma Data

DECEMBER 13, 2020

2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development.

Clinical Development 40

Clinical Development Clinical Trials Therapies Trials 40

FDA Law Blog: Drug Discovery

OCTOBER 2, 2023

However, the two previous FDA guidance documents, released in May 1998 ( Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products , available here ) and December 2019 ( Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products , available here ), differ from the new guidance.

FDA 64

FDA Drug Development Disease Trials 64

The Pharma Data

JANUARY 17, 2021

The companies announced in January 2019 a collaboration agreement to develop, manufacture and commercialize SPR206 in the Territory. SPR206 is in clinical development as an innovative option for the treatment of multi-drug resistant (MDR) Gram-negative bacterial infections. SHANGHAI , Jan. ” About SPR206.

Clinical Trials 52

Clinical Trials Clinical Development Pharmacokinetics Pharmaceutical Companies 52

The Pharma Data

JANUARY 7, 2021

Cerecor intends to use the net proceeds of the offering for general corporate purposes and working capital, primarily to support the ongoing clinical development of key assets within its pipeline and for general and administrative expenses. ROCKVILLE, Md. and CHESTERBROOK, Pa., 07, 2021 (GLOBE NEWSWIRE) — Cerecor Inc.

Clinical Development 52

Clinical Development Disease Treatment FDA Approval 52

The Pharma Data

NOVEMBER 23, 2020

NYSE American: SYN) is a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need. . ROCKVILLE, Md. , 24, 2020 /PRNewswire/ — Synthetic Biologics, Inc. Synthetic Biologics, Inc.

Compliance 52

Compliance Licensing Licensing Clinical Trials 52

The Pharma Data

JANUARY 3, 2021

InspirMed is a newly established subsidiary of TLC specializing in the development of inhalable liposome formulation programs, such as TLC19 for the treatment of Coronavirus disease 2019 (COVID-19) as well as other programs for severe acute and chronic pulmonary diseases. Cautionary Note on Forward-Looking Statements.

Clinical Trials 52

Clinical Trials Pharmaceutical Companies Trials Disease 52

Drug Target Review

AUGUST 24, 2023

These findings were mirrored in the clinical results from an analysis of two metastatic melanoma patient cohorts who received anti-PD-1 treatment: patients with responses and disease control had significantly lower GDF-15 serum levels (measured before treatment start), while none of the patients with elevated GDF-15 levels showed a lasting response.

Treatment 98

Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

DECEMBER 10, 2020

executive vice president of research and development. “We We look forward to working closely with the FDA throughout the clinical development process to bring this potential new innovative treatment to patients as quickly as possible.”. Safe Harbor Statement.

FDA 52

FDA Pharmaceuticals Small Molecule Clinical Development 52

The Pharma Data

JUNE 2, 2022

Affinivax is pioneering the development of a novel class of vaccines, the most advanced of which are next-generation pneumococcal vaccines. A 30-plus valent pneumococcal candidate vaccine is also in pre-clinical development. pneumoniae invasive disease and pneumonia in adults 50 years and above.

Vaccine 52

Vaccine Immune Response Clinical Trials Disease 52

The Connected Lab

APRIL 8, 2020

The cost to develop a new prescription medicine that gains market approval has gone up 145% to $2.6 billion and takes an average of 10 years to develop 1. To this day, more than 400 million people suffer from rare diseases and 95% of rare diseases lack an FDA approved treatment 3. 2019, Freedman, David H.

Pharma Companies 52

Pharma Companies Laboratories Drugs FDA Approval 52

The Pharma Data

MAY 31, 2022

Affinivax is pioneering the development of a novel class of vaccines, the most advanced of which are next-generation pneumococcal vaccines. A 30-plus valent pneumococcal candidate vaccine is also in pre-clinical development. pneumoniae invasive disease and pneumonia in adults 50 years and above. Pneumococcal disease.

Vaccine 52

Vaccine Immune Response Clinical Trials Disease 52

The Pharma Data

MARCH 2, 2021

This new collaboration builds on the positive experience from previous partnerships with Gubra and combines Gubra’s proprietary streaMLine platform and expertise in obesity and peptide chemistry with Boehringer Ingelheim’s expertise in lead optimization and clinical development. population will have obesity. About Boehringer Ingelheim.

Treatment 52

Treatment Bioinformatics Clinical Development Therapies 52

The Pharma Data

OCTOBER 31, 2020

Kuo-Lung Huang , the Chairman and CEO of TaiGen notes, “Influenza is a life threatening disease with significant unmet medical needs and high medical burden. With the IND approval in place, we are ready and eager to extend the clinical study of TG-1000 to the U.S. billion USD in 2019 and is estimated to reach 5.03

FDA Approval 52

FDA Approval FDA Clinical Development Virus 52

The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Phase1b/2 clinical study to be conducted in the United States and several European countries. Billion by 2025. and Europe.

Clinical Trials 40

Clinical Trials Science Disease Trials 40

The Pharma Data

JANUARY 24, 2021

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Research and development efforts will be led by Dr. Paul Van Slyke.

FDA 52

FDA Drugs Clinical Development Disease 52

The Pharma Data

AUGUST 1, 2021

The approval by the Food and Drug Administration (FDA) was based on efficacy and safety data from the Saphnelo clinical development programme, including two TULIP Phase III trials and the MUSE Phase II trial. Systemic lupus erythematosus SLE is an autoimmune disease in which the immune system attacks healthy tissue in the body.

Trials 52

Trials Therapies Disease Treatment 52

The Pharma Data

OCTOBER 19, 2020

Arthritis sufferers treated with upadacitinib had a significantly greater reduction in their symptoms and disease activity than people treated with a standard disease-modifying antirheumatic drug (DMARD), said co-researcher Dr. Aileen Pangan. 15 issue of the New England Journal of Medicine. ” Rinvoq received U.S. .

Clinical Trials 52

Clinical Trials Drugs Trials Treatment 52

The Pharma Data

DECEMBER 12, 2020

Dedicated rare disease unit to be headquartered in Boston. Pascal Soriot , Chief Executive Officer, AstraZeneca , said: “ Alexion has established itself as a leader in complement biology, bringing life-changing benefits to patients with rare diseases. This acquisition allows us to enhance our presence in immunology.

Regulations 75

Regulations International Disease Small Molecule 75

The Pharma Data

JUNE 2, 2022

Per 2019 agreement, Bristol Myers Squibb to also add one additional autologous TCR-T target where Immatics will receive an upfront payment of $20 million and be eligible for milestone payments and royalties. Our manufacturing process is designed to create hundreds of doses from one single donor leukapheresis. About Bristol Myers Squibb.

Therapies 52

Therapies Production Engineering Clinical Development 52

The Pharma Data

JANUARY 25, 2021

. “This marks our second strategic agreement with Asieris, a well-run and well-capitalized company that is executing ahead of plan on the clinical development of Cevira ®, which we licensed to them in July 2019. Founder and CEO of Asieris. ” About Photocure ASA.

Licensing 40

Licensing Licensing Clinical Development Pharmaceutical Companies 40

Alta Sciences

APRIL 17, 2024

Ketamine is a dissociative anesthetic approved in 2019 by the U.S. Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. It is a growing field where new technology, and an increasing understanding of the science, are bringing advancements in drug development.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

The Pharma Data

JANUARY 27, 2021

.–( BUSINESS WIRE )– PlateletBio , a pioneering biotechnology company developing next-generation allogeneic cell therapies for treatment of human diseases, has named Steven M. Altschuler, M.D., as Chairman of its Board of Directors. “As President and CEO of PlateletBio. The company, based in Watertown, Mass.,

Therapies 40

Therapies Engineering Clinical Development Disease 40

The Pharma Data

OCTOBER 9, 2021

“While we did not achieve proof of concept in this study, we are committed to advancing our deucravacitinib clinical program in inflammatory bowel disease, including ulcerative colitis and Crohn’s disease, as well as in psoriatic arthritis, lupus and other immune-mediated diseases. About Ulcerative Colitis.

Disease 52

Disease Trials Science Immune Response 52

The Pharma Data

OCTOBER 15, 2020

ASLAN believes ASLAN003 has the potential to bethe most potent oral inhibitor of DHODH currently in development for autoimmune disease, more than 30 times more potent at inhibiting the DHODH enzyme than teriflunomide. In 2019, ASLAN completed a Phase 2 study testing ASLAN003 in AML. SINGAPORE, Oct.

Pharmaceuticals 40

Pharmaceuticals Cell Based Assays Clinical Development Treatment 40

The Pharma Data

DECEMBER 2, 2020

Marstacimab is in development as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in individuals with severe hemophilia A or B (defined as factor VIII or factor IX activity <1%, respectively) with or without inhibitors. In September 2019, the U.S. About Pfizer Rare Disease. About Hemophilia.

Therapies 52

Therapies Treatment Disease Clinical Trials 52

The Pharma Data

NOVEMBER 12, 2020

The CALAVI trials were launched based on preclinical and early clinical evidence that Calquence could decrease the hyperinflammatory immune response and improve clinical outcomes in patients hospitalised with respiratory symptoms of COVID-19.1 Wilmington, DE; AstraZeneca Pharmaceuticals LP; 2019. Available online.

Trials 52

Trials Clinical Trials Immune Response FDA Approval 52

The Pharma Data

DECEMBER 29, 2020

HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the company was officially included into the Shenzhen-Hong Kong Stock Connect program (the “Hong Kong Stock Connect”).

Clinical Development 52

Clinical Development FDA Regulations Clinical Trials 52

The Pharma Data

OCTOBER 28, 2020

SIGNAL Phase 2 trial data continue to support cognitive benefit and reduced brain atrophy with pepinemab treatment in Huntington’s disease. Data provide strong rationale for continued development in Huntington’s and other slowly progressive neurodegenerative diseases, including Alzheimer’s. ROCHESTER, N.Y., Vaccinex, Inc.

Clinical Trials 40

Clinical Trials Treatment Clinical Development Disease 40

The Pharma Data

MARCH 23, 2021

For those patients with primary central nervous system (CNS) tumors and with CNS metastases, responses and a high disease control rate have been observed. We have the passion and determination to develop innovative medicines that help improve and extend the lives of people living with cancer. It is already approved in the U.S.,

Treatment 52

Treatment Clinical Trials Trials Clinical Development 52

The Pharma Data

JANUARY 13, 2021

Structure 3:1031-1039 (1995) 3 Waters J, Biocentury ; October 26, (2018) 4 NCT02273960; ClinicalTrials.gov ; “Study to Evaluate Safety and Efficacy in Adult Subjects With ITP (ITP)”; results posted April 1, 2019, updated July 29, 2019 and accessed Jan 11, 2021 5 Ferrant JL et al., International Immunol. (11):1583

Pharmaceuticals 52

Pharmaceuticals Small Molecule Vaccine Protein Expression 52

The Pharma Data

JANUARY 12, 2021

The launch of the C&GT Platform is supported by a number of collaborations and acquisitions the Germany-based company made over the past year, including the October acquisition of AskBio’s AAV-based gene therapy pipeline , as well as its 2019 deal for BlueRock Therapeutics and that company’s induced pluripotent stem cells (iPSC) platform.

Therapies 52

Therapies Licensing Licensing Pharmaceuticals 52