pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

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pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly. million price tag.

Gene Therapy 66

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Drug Discovery World

AUGUST 28, 2023

Could it be possible to prevent genetic diseases? PH: As well as starting clinical trials with our most advanced programme, SAM001, we have two more highly promising programmes that we want to advance to the clinic in the next year or two. DS: What’s next for the company’s pipeline? LinkedIn profile.

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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.

Business Development 40

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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.

Drugs 66

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Drug Discovery World

APRIL 3, 2024

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage.

Genetic Disease 52

Genetic Disease Development Research Clinical Trials 52

pharmaphorum

OCTOBER 5, 2022

” Financial terms of the agreement have also not been disclosed, but from a logistical perspective TrialSpark will take the lead on clinical development, in consultation with Sanofi. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 105

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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.

Gene Therapy 95

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Drug Discovery World

NOVEMBER 17, 2022

The use of personalised risk information allows the identification and invitation of participants to take part in future research studies which will enable more targeted and hopefully efficient clinical development programmes. Improving representation in health research. References.

Drugs 52

Drugs Research Genetic Disease Genetics 52

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.

Gene 52

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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.

Gene Therapy 52

Gene Therapy Trials Genetics Drugs 52

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.

Trials 52

Trials Protein Gene Genetic Disease 52

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 .

RNA 52

RNA Antibody Life Science Protein 52

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.”

Clinical Trials 111

Clinical Trials Clinical Trials Trials Development 111

Drug Discovery World

JULY 11, 2022

There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Methods& clinical development, 21,524–529. Conclusion. The promise of gene therapy is substantial. 6: Drago, D., Foss-Campbell, B.,Wonnacott, Wonnacott, K., Barrett, D., &Ndu, A. Molecular therapy. link] omtm.2021.04.001.

Gene Therapy 52

Gene Therapy Gene Development Production 52

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.,

Clinical Trials 40

Clinical Trials Clinical Trials Trials Development 40

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.

RNA 105

RNA Genetics Genetic Disease Medicine 105

Drug Discovery World

FEBRUARY 28, 2023

Recent progress on mRNA therapeutics – with at least five programmes in clinical studies – is encouraging, and I hope that we will only see more scientific advancement in the years to come.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

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.

Protein 52

Protein RNA Production Genetics 52

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

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. This guidance provides clarity on the path to registration for AVXS-101 IT.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

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.

Protein 40

Protein Production Development Drugs 40

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. If the initial purchasers exercise their option to purchase additional notes in full, the Company expects to use approximately $8.0

Sales 52

Sales Marketing Development Containment 52

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.

FDA Approval 52

FDA Approval Production RNA Medicine 52

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.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52