pharmaphorum

OCTOBER 16, 2020

In the age of artificial intelligence, no trial data should be going to waste. Findacure’s Rick Thompson looks at how these technologies could bring us closer to treatments for underserved rare diseases. The repurposing of drugs is becoming more common, especially in the field of rare diseases.

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The Pharma Data

NOVEMBER 29, 2020

Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.

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pharmaphorum

DECEMBER 8, 2021

Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm a little. Results from a phase 3 trial of blarcamesine are due next year. At 12 weeks, trofinetide-treated patients had a 5.1-point

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Marketing Drugs Life Science Trials 59

The Pharma Data

NOVEMBER 8, 2020

The New Drug Application (NDA) for the drug is based on data from four Phase III trials in pediatric patient populations from the age of 6 to 17 years, two Phase II trials, several Phase I trials, a long-term open label extension study, preclinical testing, and drug manufacturing data. This is under Priority Review.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Those funds were used to advance its discovery and development programs. Harr was also a director for Loxo Oncology.

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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.

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The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Sigilon Therapeutics

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RNA Antibody Life Science Protein 52

Delveinsight

AUGUST 17, 2021

The efficacy and safety results were produced from a broad research study, multicenter, open-label, randomized phase 3 trial data from the Keynote-581 trial of the first-line therapy indicated to the patients that resulted in an extension of their survival. The rarity of disease – 1.36 person/million around the globe.

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The Pharma Data

DECEMBER 20, 2020

The submission included data from two positive pivotal Phase III trials. Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. Approval demonstrates our ability to execute upon the entirety of the drug development and registration process.

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XTalks

DECEMBER 29, 2022

Vertex is planning to submit a Biologics License Application (BLA) by the end of the first quarter of 2023 and the company says it is also on track to submit exa-cel to UK and European Union (EU) regulators by the end of the year. As such, most gene therapies in late-stage clinical trials are in oncology or rare disease indications.

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The Pharma Data

NOVEMBER 2, 2020

Although the FDA did not identify any filing issues with the NDA, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. About Achondroplasia. In the U.S.,

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pharmaphorum

OCTOBER 25, 2022

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases.

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Gene Therapy Gene Genetic Disease Genetics 104

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

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Genetics Vaccination Vaccine DNA 111

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

MARCH 6, 2024

The prevalence of these diseases has grown substantially in recent decades due to an increasing global population, longer life expectancies and increasing morbidity with predisposing risk factors. This now represents one of the most challenging and costly burdens to health and social care systems and societies worldwide.

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The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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Protein RNA Production Genetics 52

The Pharma Data

OCTOBER 15, 2020

The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. The committee is tasked with supporting BeyondSpring’s business development activities related to its lead asset, Plinabulin, and other pipeline assets. BeyondSpring – BeyondSpring Inc.

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In-Vivo Business Development Manufacturing Development 52

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.

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Drug Discovery World

FEBRUARY 22, 2024

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

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