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BridgeBio has been highly active in the geneticdiseases 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.
Additionally, Astellas will obtain an exclusive option for licencing two clinical-stage programmes of Taysha, namely TSHA-102 and TSHA-120, for Rett syndrome and GAN, respectively. Astellas will also receive specific rights linked to any possible change of Taysha’s control.
The BGTC aims to expedite the development, manufacture and delivery of customised or “bespoke” gene therapies to treat rare diseases, including some which are too rare to be of commercial interest. The collaboration creates a reusable standardised method that minimises upfront costs and helps to reduce development barriers.
Merck will also make royalty payments on any approved products developed out of the partnership. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and geneticdisease areas.
When developing a clinicaldevelopment 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.
A tiny child with a devastating geneticdisease 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 ClinicalDevelopment , is available for free download. “They called him ‘The Boy.’
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
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 geneticdiseases with a focus on blinding disorders of the retina.
Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current geneticdisease 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.
” Financial terms of the agreement have also not been disclosed, but from a logistical perspective TrialSpark will take the lead on clinicaldevelopment, in consultation with Sanofi. The aim will be to identify promising candidates in areas of “unmet patient need.”
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 clinicaldevelopment, and AZ says it intends to start clinical testing of Dogma’s candidate next year.
Dr. Kehua Fan has over 15 years of Business Development, ClinicalDevelopment 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.
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 geneticdisease that causes cardiac and central nervous system dysfunction.
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 geneticdiseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.
Minimising any of those delays that happen throughout the clinical trial, or the clinicaldevelopment is really important and, more and more, it’s about having experts involved in providing advice in that clinicaldevelopment and clinical trial programme [that] is really important.”
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 clinicaldevelopment.
The submission includes data from the ENLIGHTEN clinicaldevelopment program in schizophrenia, and pharmacokinetic (PK) bridging data comparing ALKS 3831 and Zyprexa (olanzapine), to support an indication for schizophrenia.
Now poised to advance a robust therapeutics pipeline to clinicaldevelopment, 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 .
Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-geneticdiseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.
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.,
PH1 is an ultra-rare geneticdisease 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.
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized geneticdiseases at their source. If the initial purchasers exercise their option to purchase additional notes in full, the Company expects to use approximately $8.0
Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare DiseaseClinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their geneticdiseases.
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare geneticdiseases. GeneTx licensed the rights to antisense technology intellectual property from the Texas A&M University System in December 2017. About Ultragenyx.
Dr. Newman also said that as a first-in-class RNA-targeted medicine, Ionis believes donidalorsen could potentially advance the prophylactic treatment paradigm for HAE.
Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinicaldevelopment 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.
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare geneticdiseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinicaldevelopment across various therapeutic areas. While these therapies span all stages of clinicaldevelopment, they are particularly concentrated in earlier phases, indicating strong future growth.
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