Targeting brain delivery of lipid and polymer nanoparticle-based gene therapies with Sunshine

Live30 webinars are thirty-minute presentations designed to update you on the latest innovations, applications, and data in a fast yet interactive format.

Problem

Delivering therapeutic agents to the brain remains a critical challenge in the treatment of neurological diseases, particularly for gene therapies. The blood–brain barrier (BBB) severely restricts the entry of therapeutics, especially macromolecules like nucleic acids, into the central nervous system (CNS). While viral vectors can bypass the BBB, their use is limited by factors such as immunogenicity, restricted cargo capacity, manufacturing complexity, and safety concerns. There is, therefore, a significant unmet need for the development of non-viral delivery platforms capable of enabling safe, efficient, and targeted delivery of gene therapies to the brain.

Solution

The Joshi Lab at Brigham and Women’s Hospital and Harvard Medical School is developing both lipid- and polymer-based nanoparticle platforms for brain-targeted delivery of gene therapies. Achieving efficient and reproducible brain delivery requires careful optimization of formulation parameters, solvent compatibility, and processing conditions. To support this effort, the lab utilizes the Sunshine instrument from Unchained Labs, an automated microfluidic formulation system that enables high-throughput screening of nanoparticle formulations across a wide range of materials and solvents. Sunshine significantly reduces hands-on time while improving reproducibility and offering greater flexibility in formulation design.

Scope of the Seminar

In this webinar, Dr Ben Knappett will introduce the Sunshine instrument, and Dr Nitin Joshi’s team will share results from their brain-targeting studies, including formulation strategies, particle characterization data, and in vivo biodistribution outcomes. Attendees will gain practical insights into the development of brain-directed delivery platforms using both lipid and hybrid nanoparticles.

• Insight into the challenges of brain targeting
• Exclusive updates from Dr Joshi’s team
• Application of Sunshine for challenging particle formulations
• Boost in development throughput with Sunshine
• Strategic approaches for delivery of gene therapies for neurolgical diseases

Benjamin Knappett

Product Manager at Unchained Labs

Ben Knappett is Product Manager for the Sunny Suite, Unchained Labs’ automated microfluidic nanoparticle synthesis instruments, used primarily for LNP encapsulation in drug delivery, vaccines, and gene therapy applications. Ben received his MChem degree from Durham University, and completed a PhD in nanoparticle synthesis and characterization at the University of Cambridge. Ben previously worked for Particle Works as Head of Science and Applications, playing a key role in the development and commercialization of the instruments that now make up the Sunny Suite.

Nutan Bhingaradiya

Postdoctoral Fellow & Principal Investigator at Brigham and Women's Hospital, Harvard Medical School & The Joshi Lab

Dr Nutan Bhingaradiya is a postdoctoral fellow in the Department of Anesthesiology, Perioperative, and Pain Medicine at Brigham and Women’s Hospital, Boston, USA, working under the mentorship of Dr Nitin Joshi. She completed her PhD in Chemical Science from the Academy of Scientific and Innovative Research, CSIR-CSMCRI Bhavnagar, India, where her research focused on biodegradable amphiphilic copolymers and conetworks for biomedical applications. Dr Bhingaradiya has expertise in polymer chemistry, drug delivery systems, and biomaterials, with a particular interest in injectable hydrogels, micellar formulations, and hemostatic agents for wound healing.

Mahima Dewani

Postdoctoral Fellow & Principal Investigator at Brigham and Women's Hospital, Harvard Medical School & The Joshi Lab

Dr Mahima Dewani completed her PhD from the Indian Institute of Technology, Bombay, India, and is currently a postdoctoral fellow at Brigham and Women's Hospital, Harvard Medical School, Boston. Dr Dewani was a silver medalist during her bachelor's and received the prestigious Prime Minister’s Research Fellowship during her PhD Her work focuses on the design of biomaterials for targeted drug delivery and has been published in multiple high-impact journals, including Biomaterials, PNAS, and ACS Bioconjugate Chemistry.

Watch now

SPEAKERS

Benjamin Knappett

Product Manager at Unchained Labs

Nutan Bhingaradiya

Postdoctoral Fellow & Principal Investigator at Brigham and Women's Hospital, Harvard Medical School & The Joshi Lab

Mahima Dewani

Postdoctoral Fellow & Principal Investigator at Brigham and Women's Hospital, Harvard Medical School & The Joshi Lab

You might also like

Exploring the advantages of ready-to-use lipid-based gene delivery for gene-modified cell therapy

Chesney Michels, Claire Guéguen

25 June

Watch

Interferometric light microscopy for rapid virus titering and characterization of lipid nanoparticle preparations 

Ben Josey

31 October 2024

Watch

Elevate LNP optimization with Sunshine and Stunner

Kevin Lance

17 October 2024

Watch

Combining AI-powered mRNA design with non-viral delivery to achieve organ-specific targeting

Claire Guéguen, Davide De Lucrezia

15 July

Watch