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

The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. VLA15 has demonstrated strong immunogenicity and safety data in pre-clinical and clinical studies so far.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

Drug Target Review

APRIL 3, 2024

Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. in North America) in late 2017. Everything started in school with an experiment on isolating DNA from bananas.

Science 105

Science Clinical Development Treatment DNA 105

The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

Disease 40

Disease FDA Treatment Therapies 40

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

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

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

JUNE 25, 2021

protofibril antibody for the treatment of Alzheimer’s disease (AD). Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. Lecanemab selectively binds to neutralize and eliminate soluble, toxic amyloid-beta (A?)

Disease 40

Disease Treatment Therapies FDA 40

The Pharma Data

MARCH 1, 2022

We are very appreciative of the trust that Astellas placed in Affinivax and our novel MAPS technology platform when we signed our original agreement in 2017, and their commitment to our partnership since then.”. The FDA decision is informed by the results of the Phase 2 clinical data. Brugger, CEO of Affinivax. “We

Vaccine 52

Vaccine Clinical Trials FDA Therapies 52

The Pharma Data

JANUARY 10, 2021

About MorphoSys MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for patients suffering from cancer and autoimmune diseases.

Clinical Trials 52

Clinical Trials Clinical Development Clinical Supply Trials 52

The Pharma Data

MARCH 1, 2022

The mechanism is currently being evaluated for the potential treatment of cognitive impairment and other symptoms associated with a range of neuropsychiatric and neurodegenerative disorders, such as Alzheimer’s disease and major depressive disorder. senior vice president, R&D, chief scientific officer, AbbVie.

Small Molecule 52

Small Molecule Licensing Licensing Disease 52

Alta Sciences

APRIL 17, 2024

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. Read The Altascientist , “Nonclinical Studies in Cell and Gene Therapy”. 3.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

The Pharma Data

DECEMBER 3, 2020

Based on the positive Phase 1 results reported and the unprecedented need for COVID-19 vaccines, Clover and its partners are confident to enter late-stage clinical development for both adjuvanted vaccines. The Phase 1 clinical trial was funded by the Coalition for Epidemic Preparedness Innovations (CEPI).

Vaccine 52

Vaccine Immune Response Virus RNA 52

The Pharma Data

APRIL 4, 2022

Dupixent was also granted Orphan Drug designation for the potential treatment of EoE in 2017. The potential use of Dupixent in EoE is currently under clinical development, and the safety and efficacy have not been fully evaluated by any regulatory authority. Regulatory filings around the world are also planned in 2022.

FDA 52

FDA Treatment Disease FDA Approval 52

The Pharma Data

DECEMBER 8, 2020

Some of the funds will be used for the development of therapeutics for oncology and other serious diseases through its proprietary ImmunoTAC technology platform. The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites.

RNA 52

RNA Therapies Disease Protein Expression 52

The Pharma Data

JULY 29, 2021

Food and Drug Administration (FDA) in June 2021 — following 18 months of treatment in the open-label extension (OLE) of the Phase 2b proof-of-concept study in subjects with early Alzheimer’s disease (AD) (Mild Cognitive Impairment [MCI] due to AD and mild AD) at the Alzheimer’s Association International Conference (AAIC) held in Denver, Colo.,

Clinical Trials 52

Clinical Trials Treatment Clinical Development Disease 52

The Pharma Data

JANUARY 12, 2021

The roots of Bayer’s platform began in 2014, when the company stepped into the gene therapy space through a collaboration for hemophilia A with Dimension Therapeutics, which was later acquired by Ultragenyx in 2017. There is an incredible unmet need in some diseases that can only be addressed through cell and gene therapies.”.

Therapies 52

Therapies Licensing Licensing Pharmaceuticals 52

The Pharma Data

JANUARY 3, 2021

Nasdaq: INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced that the U.S. We are initially focused on developing a novel therapy to treat ENPP1 and ABCC6 deficiencies.

Clinical Trials 52

Clinical Trials Trials Treatment FDA 52

The Pharma Data

JANUARY 31, 2021

These findings give confidence that Clover’s COVID-19 vaccine candidates are suitable for further clinical development.” Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. . CHENGDU, China , Feb. About Trimer-Tag© Technology.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

The Pharma Data

NOVEMBER 11, 2020

McChesney is the founder and chief executive officer of Cloaked Therapeutics and Arbor Therapeutics, through which he has drawn on more than five decades of research in natural products chemistry to identify and develop novel cancer treatments. a consulting and research firm where he acted as the principal until 2017.

Organic Chemistry 52

Organic Chemistry Science Pharmacy Pharmaceuticals 52

The Pharma Data

MAY 31, 2022

Without treatment, infants do not achieve these milestones in the natural history of the disease. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Food and Drug Administration in 2017.

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

The Pharma Data

NOVEMBER 1, 2020

Highly innovative first-in-class study for treating COVID-19 disease with nasally administered drug. Clinical Data Expected by End of Year. This scientific advancement provides the basis to move forward with clinical development of nasally administered Foralumab in COVID-19 disease.”

Clinical Trials 40

Clinical Trials Trials Medical Schools Hospitals 40

The Pharma Data

JANUARY 3, 2021

Preclinical data from our laboratory have shown that the nasal administration of anti-CD3 stimulates Tregs that can suppress inflammation and ameliorate inflammatory diseases. Patient reported outcome to assess clinical responses related to COVID-19 symptoms, as per the FDA guidelines, will also be collected.

Clinical Trials 40

Clinical Trials Science Treatment Trials 40

The Pharma Data

DECEMBER 17, 2020

Cadavid will be responsible for the strategy, direction and execution of the company’s clinical development programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinical development group.

Small Molecule 52

Small Molecule Engineering Clinical Development Pharmaceuticals 52

The Pharma Data

APRIL 29, 2022

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Food and Drug Administration in 2017. About SMA SMA is a severe, progressive neuromuscular disease that can be fatal. The study met its primary endpoint.

Treatment 52

Treatment Clinical Trials Trials Clinical Development 52

Metabolite Tales Blog

JANUARY 23, 2024

The drug is therefore contraindicated in cases where severe renal impairment or end‐stage renal disease has been confirmed, as well as in combination with CYP1A2 inhibitors [7]. Pharmacokinetics and Disposition of Momelotinib Revealed a Disproportionate Human Metabolite—Resolution for Clinical Development. link] [13] Eng et al.,

Small Molecule 52

Small Molecule FDA Approval FDA Pharmacokinetics 52

LifeSciVC

APRIL 27, 2023

Fourteen years on, this corporate experiment has gone far beyond the initial idea, and has established an R&D engine more effective than most big pharma R&D groups at producing best-in-class small molecules against targets that matter in human disease biology. and swiveled her monitor to show me the news.

Small Molecule 40

Small Molecule Computational Chemistry Engineering Clinical Development 40

The Pharma Data

DECEMBER 21, 2020

Targeted protein degradation (TPD) is an approach whereby the body’s natural process for degrading proteins is diverted using small molecule drugs to eliminate disease-causing proteins. We are delighted to enter this collaboration with Captor, which has developed multiple novel approaches to identify small molecules that promote TPD.

Targeted Protein Degradation 40

Targeted Protein Degradation Small Molecule Structure Based Drug Design (SBDD) Drugs 40

The Pharma Data

NOVEMBER 11, 2020

. Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S.

Clinical Trials 40

Clinical Trials Trials Disease FDA 40

The Pharma Data

JUNE 30, 2021

Event-free survival is defined as time from randomization to disease progression, commencement of new lymphoma therapy, or death from any cause. Food and Drug Administration (FDA) whereby the trial design, clinical endpoints and statistical analysis were agreed in advance with the Agency. “As This press release features multimedia.

Therapies 52

Therapies Treatment Virus Vaccine 52

The Pharma Data

NOVEMBER 5, 2020

Taveras will lead all research and non-clinical development functions supporting the company’s pipeline of investigational therapies. Prior to Akcea, she was senior vice president and General Manager of Cardiometabolic and Rare Disease Businesses and Strategic Alliances at Moderna, Inc. Schijns, a former professor at N.C.

Analytical Chemistry 40

Analytical Chemistry Pharmaceuticals Small Molecule Therapies 40

The Pharma Data

OCTOBER 28, 2020

(NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40

The Pharma Data

NOVEMBER 8, 2020

In 2017 we bet on refocusing our strategy on medical dermatology to best address patients’ unmet needs. Likewise, we continue to build an R&D pipeline to make a significant difference in patients’ lives by treating a wide range of skin diseases. ” Peter Guenter , CEO.

Licensing 52

Licensing Licensing Production Treatment 52

The Pharma Data

JULY 23, 2021

Opdivo was first granted this indication in 2017 under the FDA’s accelerated approval program, making it the first immunotherapy agent to be approved for use in this setting. Opdivo helped usher in an entirely new way to treat patients with this disease. Opdivo helped usher in an entirely new way to treat patients with this disease.

Treatment 52

Treatment Trials Therapies Disease 52

The Pharma Data

SEPTEMBER 15, 2020

Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology. In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Rare Disease. BNT162 mRNA-based Vaccine Program.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

The Pharma Data

APRIL 1, 2021

In 2017, Tecartus was granted Breakthrough Therapy Designation by the FDA for relapsed or refractory adult B-cell precursor ALL. 18 years old) with ALL whose disease is refractory to or has relapsed following standard systemic therapy or hematopoietic stem cell transplantation. About ZUMA-3.

Treatment 52

Treatment Virus Therapies FDA 52

Agency IQ

AUGUST 18, 2023

BY COREY JASEPH, MS, RACAUG 11, 2023 4:47 PM CDT Quick refresher on the European regulations Europe enacted the medical device and IVD regulations in May 2017 but gave manufacturers and other stakeholders, including the regulators themselves, time to implement them.

Science 40

Science Regulations Laboratories Production 40