Pitch & Partner
Pitch and Partner, Wednesday 24 April 2024
Pitch and Partner
11:25Venture capital panel: Charting the path for rare disease therapies innovation: Unveiling opportunities, trends, and challenges
Moderator: Ken Kengatharan, Managing General Partner, Atheneos Ventures
Geeta Vemuri, Managing Partner, Agent Capital
Pitch and Partner
12:25A first-in-class revolutionary platform: jCell® Retinal Progenitor Cells (RPC) restoring vision in patients with blinding diseases
John Sholar, Chief Executive Officer, jCyte Inc
Pitch and Partner
12:35Innovative biologics delivery for rare disease
Pitch and Partner
12:45Adaptable decoys for the quick and selective removal of anti-drug and anti-vector antibodies from circulation
Pitch and Partner
12:55TransCytos' Non-Viral Ex-Vivo Slipstream™ Technology – a Transformation of Cell and Gene Therapy Delivery
Pitch and Partner
13:05From patient organization to biotech
Pitch and Partner
13:15Metabolic replacement therapy for the treatment of rhizomelic chondrodysplasia punctata
Pitch and Partner
15:00Revolutionizing vision restoration: Mutation-independent optogenetic therapy for inherited retinal diseases
Pitch and Partner
15:10How to prepare for the genomic gold rush in pharma
Pitch and Partner
15:20ST-002: A promising therapy for the metabolic effects of hypercortisolemia and Cushing syndrome
Pitch and Partner
15:30Zelenirstat: Safety and efficacy - ready to move to clinical investigation for Acute Myeloid Leukemia (AML)
Pitch and Partner
15:40Long acting glucagon analog for the treatment of CHI (Congenital Hyper Insulinism)
Pitch and Partner
15:50FLAG-003 for the Treatment of DIPG (Diffuse Intrinsic Pontine Glioma) in Children
Pitch and Partner
16:00First-in-class KCC2 potentiators as novel orphan drugs to treat CNS disinhibition and drug-resistance in rare neurological disorders
Pitch and Partner
16:10NPC, Galactosemia and Barth Syndrome: Developing modifier therapies for neurological and metabolic disorders
Pitch and Partner
16:20Ensuring reliability in quantifying patient populations for orphan drugs: challenges and solutions
Pitch and Partner
16:30XDemics: Making tomorrow’s cell and gene therapies
Colin Cook, Technical Founder, XDemics Corporation
last published: 25/Apr/24 16:35
Pitch and Partner, Thursday 25 April 2024
Pitch and Partner
12:00Industry perspectives: Partnering in the rare disease space
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen
Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.
Pitch and Partner
12:40Development of an enzyme replacement therapy for batten disease CLN1 and other neurodegenerative diseases
Pitch and Partner
12:50Decoding brain disease to reimagine drug development
Pitch and Partner
13:00Supercharging rare patient groups with technology
Sponsored by Comend
Albert Wang, Co-founder, CEO, Comend
Pitch and Partner
13:10A first-in-class revolutionary platform: jCell® Retinal Progenitor Cells (RPC) restoring vision in patients with blinding diseases
John Sholar, Chief Executive Officer, jCyte Inc
Pitch and Partner
13:20A-Synaptic’s patent protected transdermal replacement for billion dollar oral childhood epilepsy treatment
Pitch and Partner
15:15A candid conversation: What rare really needs
last published: 25/Apr/24 16:35
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