pharmaphorum

SEPTEMBER 17, 2020

The UK big pharma hasn’t revealed how much it is paying for rights to the drug candidate, originally developed by US biotech Dogma Therapeutics, which is still in preclinical development. At the moment no oral PCSK9 drugs have started clinical development, and AZ says it intends to start clinical testing of Dogma’s candidate next year.

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The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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The Pharma Data

OCTOBER 27, 2020

AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction.

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The Pharma Data

DECEMBER 13, 2020

Dr. Kehua Fan has over 15 years of Business Development, Clinical Development of innovative drugs and other healthcare industry experience with MNCs and biotech companies. He received his B.S. degree from Henan Normal University, M.S. degree from Chinese Academy of Sciences and Ph.D. from University of New Hampshire.

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pharmaphorum

OCTOBER 27, 2020

The disappointing result comes as three potential gene therapies for DMD that could tackle the underlying genetic cause of the disorder are making their way through clinical development.

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XTalks

NOVEMBER 26, 2024

BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

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The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. The impact of CNS diseases extends beyond patients—to their families and society as well.” Sigilon Therapeutics .

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Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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The Pharma Data

NOVEMBER 11, 2020

Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. H1 2021: Initiation of Phase 1/2 clinical trial. Demetrios Braddock, M.D.,

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The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

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The Pharma Data

OCTOBER 28, 2020

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. GeneTx licensed the rights to antisense technology intellectual property from the Texas A&M University System in December 2017. About Ultragenyx.

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XTalks

FEBRUARY 24, 2025

The data from the pivotal Phase III OASIS and OASISplus studies and three-year data from a Phase II open-label extension study show significant and clinically meaningful improvements in quality-of-life measures.

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. The Xtalks White Paper, The Intersection of Rare Disease and Advanced Therapies: What it Means for Clinical Development , is available for free download. “They called him ‘The Boy.’

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death.

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The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Oxlumo should be administered by a healthcare professional.

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The Pharma Data

NOVEMBER 8, 2020

The submission includes data from the ENLIGHTEN clinical development program in schizophrenia, and pharmacokinetic (PK) bridging data comparing ALKS 3831 and Zyprexa (olanzapine), to support an indication for schizophrenia.

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pharmaphorum

JANUARY 27, 2023

Minimising any of those delays that happen throughout the clinical trial, or the clinical development is really important and, more and more, it’s about having experts involved in providing advice in that clinical development and clinical trial programme [that] is really important.”

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The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

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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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XTalks

JANUARY 6, 2025

As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas. While these therapies span all stages of clinical development, they are particularly concentrated in earlier phases, indicating strong future growth. percent from 2022 to 2030.

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