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. Food and Drug Administration (FDA). Born with spinal muscular atrophy (SMA), the little boy was saved by the drug Zolgensma, a one-time intravenous infusion that restored the child’s defective SMN1 gene.

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Drug Discovery World

JULY 12, 2022

Destiny Pharma will establish a research project with the Cystic Fibrosis Foundation where it will establish the potential of its XF-73 drug as a treatment for MRSA infected cystic fibrosis patients. . Cystic fibrosis (CF) is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe.

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Drug Discovery World

AUGUST 28, 2023

And we’ve shown that, at least in animal models of these diseases, our drug molecules work really well. DS: Your research has so far focused on Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS). PH : This is a relatively new area of biology and it takes many years to get a new drug to the market.

Genetics 52

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pharmaphorum

OCTOBER 5, 2022

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. .” billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

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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.

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The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( He received his B.S.

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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.

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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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pharmaphorum

OCTOBER 27, 2020

Catabasis has conceded defeat with its Duchenne muscular dystrophy drug edasalonexent, pulling the plug on the drug after a phase 3 trial echoed the results of a failed mid-stage study. We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial,” said Jill Milne, Catabasis’ CEO. “I

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Gene Therapy Trials Genetics Drugs 52

The Pharma Data

OCTOBER 27, 2020

The company plans to tackle the existing challenges of precision medicine with a three-prong approach – developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs.

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The Pharma Data

NOVEMBER 8, 2020

Food and Drug Administration (FDA) in terms of PDUFA dates. The drug is an investigational, novel, once-daily, oral atypical antipsychotic drug candidate designed to improve the efficacy of olanzapine while decreasing olanzapine-associated weight gain. November is promising to be a busy month for the U.S.

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pharmaphorum

JANUARY 27, 2023

Late last year, pharmaphorum caught up with Dr Karen Mullen, chief medical officer and VP of clinical & medical affairs at global drug development consultancy Boyds. We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said. But who is benefitting? We know that there are 3.5

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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.

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The Pharma Data

NOVEMBER 11, 2020

“ENPP1 deficiency is a systemic, progressive and continuous disease occurring over the course of a patient’s lifetime, starting as early as fetal development and spanning into adulthood. Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Upcoming Investor Conference.

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Drug Discovery World

JULY 11, 2022

There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. Food and Drug Administration.[link]. Conclusion. and Peter Marks, M.D., link] omtm.2021.04.001.

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Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience. This is wholly unsurprising.

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Drug Discovery World

FEBRUARY 28, 2023

Given its recent success, I believe we will see continued innovation in the field that extends into mRNA-based therapeutics for several diseases, especially those that have had limited or ineffective drug options so far. Rare diseases often have few, if any, therapeutic options for their condition.

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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. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.

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The Pharma Data

OCTOBER 28, 2020

These SAEs were assessed as mild or moderate in severity and have generally improved over a period of a few weeks while disease domain improvements have been sustained for three months. The administration method will be modified to help reduce local drug contact time. Food and Drug Administration (FDA). About Ultragenyx.

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The Pharma Data

NOVEMBER 24, 2020

Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

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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. Source link.

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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. About BridgeBio.

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The Pharma Data

SEPTEMBER 22, 2020

Basel, September 23 , 2020 – Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (FDA) following their review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy (SMA). More than 30% of patients with SMA Type 2 will die by age 25.

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