The BioCentury Show

Arrowhead Pharmaceuticals can now deliver siRNA to seven cell types, and new data showing subcutaneous delivery to the brain could push RNAi therapies well beyond the liver. On this edition of The BioCentury Show, Arrowhead Chairman, President, and CEO Chris Anzalone joins BioCentury’s Stephen Hansen to discuss where siRNA has the edge over ASOs, what the Phase III miss for Novartis’ pelacarsen means for olpasiran, the Lp(a) therapy Arrowhead partnered with Amgen, and the growth outlook for plozasiran in severe hypertriglyceridemia. Anzalone also describes Arrowhead’s obesity programs targeting INHBE and ALK7, and a dimer molecule that silences two genes at once.

View full story: https://www.biocentury.com/article/661194

#RNAi #siRNA #DrugDelivery #DrugDevelopment #Biotech

0:00 Introduction
1:09 Arrowhead's siRNA Platform
9:05 Solving for Extra-Hepatic Delivery
23:43 Redemplo's Future Growth
27:00 Obesity and Bispecific siRNA

Untapped multibillion-dollar markets are hiding in plain sight where patients endure mediocre generic standards of care, according to Canaan General Partner Julie Grant, co-founder and first CEO of Day One, which Servier acquired for $2.5 billion this year. In an interview with BioCentury's Lindsay Martin, Grant discussed white spaces in women's health such as migraine and anemia, and why mid-tier acquirers like Jazz, Ipsen and Servier make smaller markets viable. She also explained why the last private round can understate value by 10-20X, how Canaan builds management teams around a key individual who sets the tone and why biotech needs scientific heroes as AI captures most venture dollars.

View full story: https://www.biocentury.com/article/660963

#VentureCapital #Biotech #WomensHealth #DrugDevelopment #BiotechMA

0:00 Introduction
1:19 Canaan's Strategy
11:34 Team Building
21:57 White Space
28:00 New Biotech Buyers
34:15 Biotech Founders

On this edition of The BioCentury Show, Nick Galakatos, Global Head of Life Sciences at Blackstone, joins Editor in Chief Simone Fishburn to discuss the path from Clarus to Blackstone's new $6.3 billion fund, and the goal of game-changing bets.
Galakatos discusses Blackstone's thesis for addressing the late-stage financing gap, as Phase III programs costing more than $1 billion drive demand for large, product-based capital. He also addresses structured capital as an alternative to acquisition, noting deals such as Blackstone's with Alnylam and Apogee can reduce dilution and extend runway.

View full story: https://www.biocentury.com/article/660824

#Biotech #PrivateEquity #DrugDevelopment #LifeSciences #ClinicalTrials

0:00 Introduction
1:22 New Fund & Founding
10:30 Alnylam Deal
17:14 Working with Big Caps
25:54 Biotech Risk
29:13 China

On This Edition of The BioCentury Show, Gonçalo Abecasis, SVP and Chief Genomics and Data Sciences Officer at the Regeneron Genetics Center, joins BioCentury’s Tierney Baum to discuss genetics as a scalable drug discovery platform and the practical use of AI in genomics.

View full story: https://www.biocentury.com/article/660697

#Genomics #DrugDiscovery #AI #GeneticMedicines #ClinicalTrials 

0:00 - Introduction
1:14 - Regeneron Genetics Center
10:29 - Acting on Genetic Signals
16:57 - Getting to Drug Development
21:51 - Best AI Use Cases

The Critical Path Institute’s One to Millions Initiative is using individualized rare disease therapies as the starting point for a development framework that could eventually extend across all genetically defined diseases. The initiative seeks to bring form to FDA’s Plausible Mechanism Framework by standardizing how evidence and methods can be reused across therapies and patients.
In conversation with Biopharma Analyst Dr. Tierney Baum on The BioCentury Show, C-Path CEO and CSO Klaus Romero discusses how the principal bottleneck is no longer the ability to design therapies for individual patients but rather the absence of a standardized regulatory and development system that can translate one success into many. C-Path plans to bring companies, academics, patient groups, and regulators together to share data and address recurring development bottlenecks.

View full story: https://www.biocentury.com/article/660583

#GeneticMedicines #RareDisease #DrugDevelopment #RegulatoryScience #ClinicalTrials

00:00 - Introduction
01:44 - Current Framework
07:01 - Process-based Regulation
13:57 - Overcoming Bottlenecks
20:28 - Use of AI
25:25 - Beyond Rare Diseases