Commercialization is a tough road to navigate even for the most established bio/pharmaceutical therapies. But as cell and gene therapies (CGTs) continue to form roots and make a splash worldwide—but specifically in the US—commercialization forms its own version of standardization and best practices.  

In this exclusive interview with Pharmaceutical Executive, Lung-I Cheng, vice president of CGT, Cencora (formerly AmerisourceBergen), discusses: 
1) defining CGT commercialization strategies and foundational knowledge—and the best music metaphor
2) what he wishes companies knew about commercialization strategies
3) common knowledge gaps of CGTs
4) how CGT developers can overcome key challenges, including those related to patient access, regulatory requirements, and market access
5) how planning for allogeneic cell therapies differs from autologous cell therapies and gene therapies
6) how companies can demonstrate CGT value as more products enter the development pipeline (and competition intensifies)
7) when to start planning a CGT commercialization strategy
8) predictions for the CGT market in five years

Avoiding Commercialization Pitfalls for CGTs

Ocugen Reports Updated Results for Clinical Trial of Modifier Gene Therapy

In this issue, we review the key considerations and regulatory guidance for nonclinical cell and gene therapy development, including expert approaches to mitigating complex challenges, improving study efficiency, and maximizing translational opportunities to first-in-human trials.

CHAPTERS: 
- 00:07 — About This Audiobook
- 01:57 — Introduction
- 05:34 — Species Selection 
- 09:00 — Germline Mitigation Studies
- 13:22 — Planning Your Nonclinical Studies
- 14:44 — Case Study 1
- 17:18 — Case Study 2
- 19:08 — Conclusion

Click here to read Issue 36: https://www.altasciences.com/sites/default/files/2023-09/The-Altascientist-Issue36-Cell-and-Gene-Therapy.pdf

Traditional approaches modulate the course of disease but do not provide a cure, particularly in the case of monogenic diseases, which are caused by mutations in single genes that a person is born with. It is estimated that there are greater than 6,000 monogenic diseases, affecting over 350 million people worldwide. For these diseases, cell and gene therapy may provide hope for a cure.

There are significant challenges associated with the successful development of these complex, leading-edge therapies. Challenges involved in the in vivo preclinical study of cell and gene therapies include understanding on- and off-target activity, immune responses, and other serious adverse events. All of these must be carefully monitored, rigorously assessed, and managed to the highest extent possible.

About Altasciences:
Altasciences is an integrated drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services. For over 25 years, Altasciences has been partnering with sponsors to help support educated, faster, and more complete early drug development decisions. Altasciences’ integrated, full-service solutions include preclinical safety testing, clinical pharmacology and proof of concept, bioanalysis, program management, medical writing, biostatistics, clinical monitoring, and data management, all customizable to specific sponsor requirements. Altasciences helps sponsors get better drugs to the people who need them, faster.

Issue 36 — Nonclinical Studies in Cell and Gene Therapy

In this issue, we will review the applications of PCR analysis for your gene therapy programs, and present two case studies.

Click to read this audiobook: https://www.altasciences.com/sites/default/files/2023-10/the-altascientist-issue-37-pcr_4.pdf 

Gene therapy continues to accelerate through preclinical and clinical research arenas. These programs are developed with targeted and personalized medicines in mind. The goal of gene therapy is to safely deliver and incorporate a genetic alteration to restore or repair the protein of a missing or faulty gene. Preclinical assessments of gene therapies consider the general absorption, distribution, metabolism, and excretion of the drug, as well as data from tailored assessments to evaluate delivery and cell incorporation. 

Gene therapies require, by design and definition, DNA and/or RNA delivery and analysis. While most ongoing research involves gene therapies being delivered in vivo via adeno-associated viral (AAV) vectors, new in vivo delivery mechanisms are on the rise, such as other types of delivery vectors including lipid nanoparticles.

CHAPTESR: 
- 0:05 — About Issue 37
- 1:20 — Introduction to qPCR, dPCR, and RT-PCR 
- 5:58 — Utility of qPCR, ddPCR, and RT-PCR 
- 8:19 — PCR Applications 
- 10:01 — Advantages and Applications 
- 17:45 — Case Study 1 
- 20:20 — Case Study 2 
- 22:47 — Conclusion

About Altasciences:

Altasciences is an integrated drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services. For over 25 years, Altasciences has been partnering with sponsors to help support educated, faster, and more complete early drug development decisions. Altasciences’ integrated, full-service solutions include preclinical safety testing, clinical pharmacology and proof of concept, bioanalysis, program management, medical writing, biostatistics, clinical monitoring, and data management, all customizable to specific sponsor requirements. Altasciences helps sponsors get better drugs to the people who need them, faster.

Issue 37 — Quantitative PCR and Droplet Digital: Analysis for Your Gene Therapy Programs

Audio roundup of selected biopharma industry content from Scrip over the past business week. In this episode: Daiichi’s huge ADC deal with Merck; mixed data for Rybrevant at ESMO; Pfizer girds for cost cuts; myeloma helps J&J’s Q3; and five key points for cell and gene Meeting on the Mesa.
https://scrip.citeline.com/SC149230/Quick-Listen-Scrips-Five-MustKnow-Things

Scrip’s Five Must-Know Things – 23 October 2023

일본 최대 제약바이오 R&D 허브와 한국 바이오벤처들이 세포유전자치료제 분야의 협업을 활발히 추진하고 있다는 Scrip 기사를 요약한 내용입니다.

https://scrip.citeline.com/SC149214/Japans-Largest-Biocluster-Korean-Ventures-To-Collaborate-For-Cell-And-Gene-Therapies

일본 최대 바이오클러스터, 한국 바이오벤처들과 CGT 협업 추진 (Korean-language podcast)

Audio roundup of selected biopharma industry content from Scrip over the past business week. In this episode: first ever gene editing FDA nod; US FTC ruffles pharma’s feathers; AstraZeneca pays to play in RSV; Syndax leukemia drug makes splash at ASH; BMS bets big on Chinese ADC.
https://scrip.citeline.com/SC149527/Quick-Listen-Scrips-Five-MustKnow-Things

Scrip’s Five Must-Know Things – 19 December 2023

This episode of VHHA's Patients Come First podcast features Dr. Wally Smith, a physician at VCU Health who specializes in treatment of Sickle Cell Disease. He joins us for a conversation about his path to medicine, his work, promising new gene therapy  treatments for the disease, and more. Send questions, comments, feedback, or guest suggestions to pcfpodcast@vhha.com or contact us on X (Twitter) (twitter.com/VirginiaHHA) or Instagram (www.instagram.com/virginiahha/) using the #PatientsComeFirst hashtag.

VHHA Patients Come First Podcast - Dr. Wally Smith

Audio roundup of selected biopharma industry content from Scrip over the past business week. In this episode: Bayer’s pipeline refresh; J&J’s selective pharma M&A approach; silver lining for Gilead’s Trodelvy miss?; paying for obesity drugs; and the story behind Vertex and CRISPR’s gene therapy breakthrough.
https://scrip.citeline.com/SC149699/Quick-Listen-Scrips-Five-MustKnow-Things

Scrip's Five Must-Know Things - 29 January 2024

Joe Selvaggi talks with neurobiologist and writer Dr. Anne Sydor about the potential for gene therapy to address deadly and debilitating diseases and how current health care models must adapt to encourage this nascent technology.

Episode 192: Genetic Therapy Revolution: Benefits and Barriers for Medicine’s New Horizon

In this enlightening episode, we are joined by Dr. Jasmina Jankicevic, the Chief Medical Officer of Innovaderm. Dr. Jankicevic is a globally recognized authority in the field of dermatology drug development, with her extensive experience spanning clinical trials and regulatory approvals. In this episode, we dive into the world of gene therapy. We will explore how gene therapy could revolutionize the field of dermatology, discuss the impact of stem cell therapy on dermatological treatments, and address the critical challenges that need to be considered when investigating gene therapy.

Episode 10 - Gene Therapy Uncovered

To mark World Haemophilia Day, we sit down with Professor Amit Nathwani of the UCL Cancer Institute to explore his journey to commercialising the groundbreaking haemophilia A treatment, Roctavian. Host Nigel Campbell is joined by Prof Nathwani and Dr. Rick Fagan, UCLB Director of Biopharm, to explore how Amit’s work looking after patients with haemophilia in the NHS instilled a mission to develop a 'one and done' vaccine type approach to the condition. Amit’s lab to market story, working with UCLB, led to the creation of Roctavian, a one-time gene therapy used for the treatment of adults with severe haemophilia A. The therapy, which was licensed to BioMarin Pharmaceutical Inc, received marketing approval in Europe in 2022 and US FDA approval in 2023, and is now available to patients commercially.

Date of episode recording: 2024-04-10T00:00:00Z
Duration: 00:23:58
Language of episode: English
Presenter: Nigel Campbell
Guests: Prof Amit Nathwani; Dr Rick Fagan
Producer: Nora Amin

Professor Amit Nathwani’s journey to create a one-shot cure for haemophilia A

Mutations in PDE6B are a leading cause of retinitis pigmentosa.

ARVO 2024 Highlight: Positive Two-Year Results from PDE6B Gene Therapy Clinical Trial

NeuShen CEO Joan Shen discusses with Brian Yang about developing CNS drugs in China, the venture's business rationale, challenges and fundraising. She also discusses two leading assets for schizophrenia and epilepsy in first-in-human studies, as well as a gene therapy collaboration for ALS with UMass's Gao Lab.
https://scrip.citeline.com/SC150328/China-Biotech-CEO-Interview-Starting-A-CNS-Venture-In-An-Economic-Downturn
Playlist: https://on.soundcloud.com/AaFyPUxfD2RiDEis5

China Biotech CEO Interview - NeuShen's Joan Huaqiong Shen

Synopsis:

Zandy Forbes, Founder, President, and CEO of MeiraGTx, joins host  Rahul Chaturvedi in this compelling episode. Zandy shares her inspiring journey from academia, where she developed a deep interest in molecular biology and genetics, to her significant role in biotech investing, and eventually leading a groundbreaking gene therapy company. She elaborates on Meira’s cutting-edge approach to genetic medicines, focusing on the development of innovative therapies for non-inherited diseases. Zandy discusses the unique challenges and opportunities that arise in the biotech sector, shedding light on the strategies Meira employs to navigate this complex landscape.

Biography:

Alexandria Forbes, Ph.D. is the President and CEO of MeiraGTx (NASDAQ: MGTX). MeiraGTx is a vertically integrated gene therapy company with core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. The company is developing innovative gene therapy products to cost effectively treat a range of serious medical disorders, with clinical programs currently in the eye, salivary gland and CNS. 

Prior to founding MeiraGTx, Dr. Forbes served as Senior Vice President of Commercial Operations at Kadmon Holdings, Inc., a biopharmaceutical company, from September 2013 to April 2015.Before joining the biotech industry, she spent 10 years as a healthcare investor at Sivik Global Partners (Argus Partners) and Meadowvale Partners. At Sivik, Dr. Forbes was responsible for investments in biotechnology, specialty pharmaceuticals and diagnostics public equities and was portfolio manager of the Sivik Global Life Science Fund, a long-biased public markets fund investing in biotechnology companies globally. During her time as a biotechnology investor, Dr. Forbes covered over 300 companies and gained expertise in biotechnology business strategies and clinical drug development as well as a wide range of disease areas. Particular areas of focus included HIV, HCV, diabetes, obesity, autoimmune disorders, cancer, Alzheimer’s disease, kidney disease and liver disease. 

Before entering the hedge fund industry, Dr. Forbes was an academic scientist studying the regulation of stem cell identity and different aspects of embryogeneis. She was a Human Frontiers/Howard Hughes postdoctoral fellow at the Skirball Institute of Biomolecular Medicine at NYU Langone Medical Center, where her research focused on cytoplasmic determinants and cell signaling pathways involved in the migration, establishment and maintenance of germ line stem cells in Drosophila melanogaster. Prior to this, Dr. Forbes was a research fellow at Duke University and at the Carnegie Institute at Johns Hopkins University where she studied the role of the hedgehog gene and its signaling pathway in the Drosophila embryo and adult. Dr. Forbes received her Ph.D. in Molecular Genetics from Oxford University, UK and attained a double first degree in Natural Sciences from Cambridge University, UK. 

Dr. Forbes serves on the Board of Directors of MeiraGTx, and as a Trustee and Director of the Selfridges Group Foundation, the European arm of the Weston Brain Institute, a charity supporting research into neurodegenerative diseases with the aim of speeding the time to the development of disease modifying treatments for these currently intractable diseases, particularly Alzheimer’s.

Transforming Gene Therapy: Exclusive Insights from Zandy Forbes, President & CEO of MeiraGTx

30. april 2025

I dag har vi med oss Sigrid Bratlie, strategisk rådgiver i Kreftforeningen og Langsikt - og ikke minst høyaktuell med bok om covid-pandemiens opphav: "Mysteriet i Wuhan". 

I dag er temaet celle-og genterapier, noe Sigrid kan mye om. Vi snakker om: Hva er det, hvilke behandlinger er godkjent, hva er flaskehalser og muligheter. Og vi snakker selvfølgelig mye om det norske forskningsmiljøet her, vårt porteføljeselskap Zelluna og; Karl-Johan Malmberg sin forskning som han i år er gitt  Kong Olav Vs kreftforskningspris av Kreftforeningen. Han for den for sitt arbeid med NK-celler, det vil si naturlig drepeceller.

Sigrid kommer tilbake i podkasten 11. juni. Da skal vi snakke om potensialet i kombinasjonen kunstig intelligens og bioteknologi, og hvilke implikasjoner det kan ha for kreftfeltet. 

Send oss gjerne spørsmål!

Episode 328: Celle- og genterapi med Sigrid Bratlie

The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial. 

ARVO 2025 Highlight: Beacon Reports Encouraging Results for Second Eyes Dosed in Clinical Trial for XLRP Gene Therapy

Part B of the trial will enroll nine adult patients as well as three pediatric patients.

ARVO 2025 Highlight: Atsena’s XLRS Gene Therapy Provides Vision and Structural Improvements in Phase 1/2 Clinical Trial

Citeline places biopharma and medical device professionals, and those who focus on these industries, at the forefront of knowledge and insight, by providing the perfect combination of news and information together with penetrating insight and analysis. Citeline is a leader in the field of healthcare industry information.

Phase Forward: Your Gateway to Dermatology and Rheumatology Clinical Research
Dive into the world of dermatology and rheumatology clinical research with Phase Forward, the podcast that brings you the latest insights, innovations, and expert advice. Powered by Indero, a leading full-service CRO, this podcast is your go-to source for everything you need to know about managing and conducting clinical trials in these fields.
Each episode features engaging interviews, thought-provoking discussions, and captivating stories from the forefront of clinical research. Whether you are a sponsor, investigator, or study coordinator, you will find valuable information and practical tips to enhance your work.
Stay ahead of the curve with updates on the newest advancements in trial design and methodology, and gain a deeper understanding of the processes and considerations in clinical research.
Don't miss out—subscribe now and join the conversation on dermatology and rheumatology clinical research with Phase Forward!

UCL (University College London) is consistently ranked among the world's top ten universities (QS World University Rankings 2010 - 2021). Our excellence extends across all academic disciplines, from one of the world's foremost centres for research and teaching in the biological sciences to world-renowned centres for architecture (UCL Bartlett), education (UCL IOE) and fine art (UCL Slade School).

UCL is London's leading multidisciplinary university, with more than 13,000 staff and 43,000 students from 150 different countries. Founded in 1826 in the heart of London, UCL was the first university in England to welcome students of any religion and the first to welcome women on equal terms with men.

UCL Minds brings together UCL’s knowledge, insights, and ideas through events, activities, and digital content open to everyone. Find out what’s on UCL Minds: www.ucl.ac.uk/minds

Radium er en podkast som utgis av RADFORSK. I studio snakker Elisabeth Andersen og Jónas Einarsson om siste nytt innen kreftforskning og utvikling av ny kreftbehandling og porteføljebedriftene til RADFORSK - alene eller sammen med gjester.

The Pharm Exec Podcast goes beyond the pages of the Pharmaceutical Executive print magazine and PharmExec.com to bring the latest commercial insights to the C-suite through in-depth interviews. Podcast episodes examine current trends, key conferences, and critical topics in the industry.

The Foundation Fighting Blindness is the driving force in the global development of treatments and cures for blinding diseases. To learn more, go to FightingBlindness.org.

Biotech2050 Podcast is a think tank chronicling the disruptions changing the biotech industry over the next 50 years.

As an end-to-end CRO/CDMO, we have expert knowledge in all early stages of drug development, and we’re sharing that knowledge with you in The Altascientist: The Audiobook. 

Each issue is a deep dive into a specific area of early-phase drug development, providing insider tips, considerations, and case studies. 


Altasciences is a drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services.

New tracks tagged #Genetherapy