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Rare Disease, Cell & Gene Therapy Weekly RoundUp
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Rare Disease, Cell & Gene Therapy Weekly RoundUp

Author: Partners4Access

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A weekly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit
48 Episodes
The debate over US drug pricing reform has reached fever pitch as the US Department of Health and Human Sciences announced its latest Medicare Part B proposal to include international reference pricing. The Partners4Access team looks at the background to this proposal; the political and patient organisation criticisms; the potential industry response and its ramifications. Presenter: Aparna KrishnanContributor: Max RexProducer: Aparna Krishnan
This week, we analyze Bluebirdbio's commercialization plans for its gene therapy Zynteglo including the new payment model and a delay in launch due to manufacturing issues. Presenter: Aparna KrishnanContributor: Joanna FernandesProducer: Aparna Krishnan
This week, the team discuss the emergence of 'drug buyer's clubs' in the UK involving Vertex's cystic fibrosis drug Orkambi. Presenter: Aparna KrishnanContributors: Sophie Schmitz, Ciaran CassidyProducer: Aparna Krishnan
This week, the team discuss Vertex's acquisition of Exonics and expansion of its collaboration with CRISPR Therapeutics. Also, we take an indepth look at the European Medicines Agency's approval of Bluebirdbio's gene therapy Zynteglo as a treatment for beta-thalassemia . Presenter: Aparna KrishnanContributor: Jack RawsonProducer: Aparna Krishnan
On our anniversary episode, the P4A team discuss the USFDA approval of Novartis' Zolgensma, a one time gene therapy for paediatric spinal muscular atrophy patients and a look back at the last 12 months of our podcasting journey. Presenter: Joanna FernandesContributor: Christina Poschen, Aparna KrishnanProducer: Aparna Krishnan
In this episode, we look at Novartis CEO Vas Narasimhan calling for a change in the US drug payment systems, arguing for new economic models to identify how much value a cure represents. Also, Italy's attempts at introducing a draft resolution to improve transparency in drug pricing at the World Health Assembly.Presenter: Aparna KrishnanContributors: Joanna Fernandes, Christina PoschenProducer: Aparna Krishnan
Weekly Roundup: May 18, 2019

Weekly Roundup: May 18, 2019


This week, the team discuss the deal between UK's National Health Service and Biogen prompting the successful reimbursement of Biogen’s Spinraza as a treatment for spinal muscular atrophy and Solid Biosciences’ clinical update on its Duchenne Muscular Dystrophy gene therapy product SGT-001.  Presenter: Aparna KrishnanContributor: Max RexProducer: Aparna Krishnan
This week, the P4A team discuss a new experimental therapy - Phage therapy or genetically engineered phages in order to treat bacterial infections and the acquisition of Theracon by Big Pharma firm Pfizer for upto $810 million.Presenter: Aparna KrishnanContributor: Ciaran CassidyProducer: Aparna Krishnan 
Weekly Roundup: May 3, 2019

Weekly Roundup: May 3, 2019


This week, we feature a special guest podcast speaker - Scott  Dorfman, chief executive officer of non-profit gene therapy developer Odylia Therapeutics on his personal journey and the future in gene therapy development. If you would like to know more about Odylia Therapeutics, please visit Presenter: Aparna KrishnanContributor: Scott Dorfman, CEO, Odylia TherapeuticsProducer: Aparna Krishnan
This week, we look at the European Federation of Pharmaceutical Industries and Associations or EFPIA’s patient wait survey and its key findings as well as news developments in the spinal muscular atrophy world relating to gene therapy Novartis’ Zolgensma and Biogen’s Spinraza.Presenter: Aparna KrishnanContributors: Sophie Schmitz, Jack RawsonProducer: Aparna Krishnan
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