XconomyPhiladelphia
The science underpinning the latest investigational cell and gene therapies is complex enough. But the rapid advance of technologies that support development of these kinds of drugs presents logistical considerations, too.
Drug development is an interesting process because it takes five to 10 years to get a drug to market and invariably, somewhere along that line, the technologys a little outdated before it even is approved, and theres new things out there that are better, said Jeffrey Castelli, chief portfolio officer and head of gene therapy at Amicus Therapeutics (NASDAQ: FOLD). You look at some of the gene therapies that are now just ready coming to market and you think, wow, thats a pretty outdated gene therapy approachbut you really get locked in as youre moving your product toward development.
Castelli was among the executives who spoke as part of a panel at Xconomys online Xcelerating Life Sciences Philadelphia forum last week.
Amicus, headquartered in Cranbury Township, NJ, operates a gene therapy R&D center in Philadelphia.To keep its therapies from becoming stale prior to commercialization, the company keeps an eye on new technologies that arise, Castelli said. But some innovations invariably surface too late to incorporate.
We really try to implement them early on, and then make sure that when we have our second wave of programs and products weve incorporated some of the innovation, he said. But you do get locked into your approach along the way, and theres more and more switching costs as you move along that pathway.
To guide that process Tom Wilton, chief business officer at Philadelphia-based cell therapy company Carisma Therapeutics, says its essential to collect and analyze data early on to guide the path of drug development.
As a relatively small company youve really got to focus the majority of your resource on pushing forward that lead program and getting it into the clinic, he said. Later this year well get a lot of data back from that first-in-human clinical study, the first time anyones ever taken an engineered macrophage into the clinic. What that should give us then is a set of criteria and priorities around what we need to bring forward in a next-generator program.
To date the companys consideration of new technologies has been perhaps a bit more opportunisticeverything from gene editing the macrophages to novel binders to different combination strategiesbut once Carisma has the initial clinical data in hand, that will narrow its focus, Wilton said.
Theres always a tendency [with a first-generation product] to say we could do this to this, we could do this to this, but you have to lock it down, you have to get into the clinic and get that data set to really understand what your priorities are and where you need to focus, and thats what were planning to do.
As experimental products move into later stages of development, another consideration arises, the panelists said: Producing them at scale.
For cell and gene therapy companies, figuring out when to lock in the space needed to make these drugs so it is available when needed for clinical trials or commercialization is difficult to determine precisely, especially given the shortage of manufacturers versed in some of the advanced technologies needed to do so.
There is a capacity shortage right now in cell and gene therapy manufacturing, said Audrey Greenberg, co-founder and executive managing director of Discovery Labs, an MLP Ventures-backed biotech coworking and incubator space in King of Prussia, PA. Theres estimates that its five times [current cumulative bioreactor volumetric capacity] now and will be 50 times in five years given the FDA pipeline and the dollars flowing into these companies.
Discovery Labs is building out a $1.1 billion gene and cell treatment manufacturing operation plus developing a contract development manufacturing organization to provide services to companies in the sector.
Image: iStock/f11photo
Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.
More here:
Timing Is an Essential Element of Cell & Gene Therapy Product Development - Xconomy
- Gene therapy: What is it and how does it work? | Live Science - September 21st, 2024
- How Does Gene Therapy Work? Types, Uses, Safety - Healthline - September 21st, 2024
- In race to make gene therapy for age-related blindness, 4D Molecular announces positive results - STAT - September 21st, 2024
- Penn gene therapy pioneer Jim Wilson explains why he's leaving - The Business Journals - September 21st, 2024
- Whats the Meaning of Cure in Gene Therapy? - Managed Healthcare Executive - September 21st, 2024
- Ori doubles down on Charles River collaboration with promising new data on its automated cell therapy platform - FiercePharma - September 21st, 2024
- Doctors cured her sickle-cell disease. So why is she still in pain? - Nature.com - September 21st, 2024
- Gene Therapy Company Increases Focus on Mesothelioma Program - Mesothelioma.net Blog - September 21st, 2024
- Sickle cell gene therapies roll out slowly : Shots - Health News - NPR - September 21st, 2024
- Patients At Last Begin Receiving Vertex-CRISPR and Bluebird Sickle Cell Gene Therapies - BioSpace - September 21st, 2024
- Beacon Therapeutics Presents 36-Month Interim Results from Phase I/2 HORIZON Trial of AGTC-501 in Patients with XLRP - PR Newswire - September 21st, 2024
- Beacons Gene Therapy Shows Continued Promise in Trial - TipRanks - September 21st, 2024
- How stem cell and gene therapies are revolutionising healthcare - Express Healthcare - September 21st, 2024
- Nanoscope Therapeutics to be Featured at Annual EUretina Congress in Barcelona - PR Newswire - September 21st, 2024
- 6-year-old Tennessee boy denied potentially life-saving gene therapy by insurance company - WCYB - September 21st, 2024
- Seeking a sickle cell cure: 12-year-old in DC is 1st patient in US to get new gene therapy - NBC Washington - May 24th, 2024
- Game-changer: The Hindu Editorial on approval for gene therapy to treat sickle cell disease and beta thalassemia - The Hindu - December 13th, 2023
- Early trials show promise for innovative gene therapy in lung cancer treatment - WJAR - October 16th, 2023
- Cell and Gene Therapy Manufacturing Quality Control Market Growing Trends and Technology Forecast to 2029 |... - SeeDance News - October 16th, 2023
- How Gene Therapy Can Cure or Treat Diseases | FDA - March 21st, 2023
- Genetic Therapies - What Are Genetic Therapies? | NHLBI, NIH - March 21st, 2023
- FDA approves novel gene therapy to treat patients with a rare form of ... - December 28th, 2022
- Gene Therapy - Discover How It Works Its Types And Applications - BYJUS - December 28th, 2022
- IVERIC bio Subsidiary Sells Assets of Gene Therapy Product Candidates for Treatment of Retinal Diseases - Marketscreener.com - December 28th, 2022
- Mustang Bio Announces Phase 1/2 Clinical Trial Data of MB-106, a First-in-Class CD20-targeted, Autologous CAR T Cell Therapy, to be Presented at 11th... - October 31st, 2022
- Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals... - October 23rd, 2022
- NIH researchers develop gene therapy for rare ciliopathy - National Institutes of Health (.gov) - September 12th, 2022
- Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today - September 12th, 2022
- Global Cancer Gene Therapy Market Report 2022: Benefits of Gene Therapy Over Conventional Therapies Driving Adoption - ResearchAndMarkets.com -... - September 12th, 2022
- As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field - The Philadelphia Inquirer - September 12th, 2022
- Myrtelle's rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European... - September 12th, 2022
- Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration - Business Wire - September 12th, 2022
- Urovant Sciences Receives Best in Category Award for Abstract Highlighting Investigational Novel Gene Therapy, URO-902, Presented at 2022... - September 12th, 2022
- The gene therapy that could transform the lives of millions - ABC News - September 12th, 2022
- Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire - September 12th, 2022
- Solving medical mysteries with genetics: The Penn Neurogenetics Therapy Center | Penn Today - Penn Today - September 12th, 2022
- George Clinical Expands China Team with New Project Director and Cell Gene Therapy Head Helen Xu - AsiaOne - September 12th, 2022
- IIT-B-Tata hosp cancer therapy trials show promising results - Hindustan Times - September 12th, 2022
- 7th International Congress of Myology Nice - EurekAlert - September 12th, 2022
- Discovery advances the potential of gene therapy to restore hearing loss - Salk Institute - August 11th, 2022
- Health and Tech: The promise of gene therapy to cure cancers - Telangana Today - August 11th, 2022
- Global Gene Therapy Market 2022-2027: High Incidence of Cancer & Other Targeted Diseases to Drive Growth - ResearchAndMarkets.com - Business Wire - August 11th, 2022
- Global Gene Therapy Market Report 2022: Type of Therapy, Gene Delivery Method, Type of Vector Used, Target Therapeutic Areas, Route of Administration... - August 11th, 2022
- Potentiation of combined p19Arf and interferon-beta cancer gene therapy through its association with doxorubicin chemotherapy | Scientific Reports -... - August 11th, 2022
- bluebird bio reveals plans to launch two gene therapies - PMLiVE - August 11th, 2022
- Taysha Gene Therapies to Release Second Quarter 2022 Financial Results and Host Conference Call and Webcast on August 11 - GlobeNewswire - August 11th, 2022
- The Alliance for Regenerative Medicine Announces the Appointment of Timothy D. Hunt as Chief Executive Officer - Yahoo Finance - August 11th, 2022
- Adverum Biotechnologies to Participate in the 2nd Annual H.C. Wainwright Ophthalmology Virtual Conference - Yahoo Finance - August 11th, 2022
- Stem Cell Therapy Global Market Report 2022: Rapid Growth in Emerging Markets & An Increase in Investments in Cell and Gene Therapies Driving... - August 11th, 2022
- OHSU advancing first-of-its-kind strategy to overcome infertility - OHSU News - August 11th, 2022
- Intracranial Therapeutic Delivery Market revenue will climb to US$ 4.2 Bn by the end of 2032 Persistence Market Research - GlobeNewswire - August 11th, 2022
- Is This Company In A Special Position Even As The COVID-19 Pandemic Affects Cell-Based Therapy Industry? - Benzinga - August 11th, 2022
- At ISCT This Week, Cell & Gene Therapy Grows Up - BioSpace - May 8th, 2022
- Tenaya Therapeutics to Present Preclinical Data on Gene Therapy Programs and Platform Capabilities at the American Society of Gene and Cell Therapy... - May 8th, 2022
- BioMarin to Present Findings from Ongoing Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the World Federation of Hemophilia... - May 8th, 2022
- Top 12 Emerging Gene and Cell Therapy Technologies Likely to Impact Patient Care Announced in Annual Disruptive Dozen from Mass General Brigham - Mass... - May 8th, 2022
- Mustang Bio Announces Data on Treatment with Lentiviral Viral Vector Gene Therapy for X-Linked Severe Combined Immunodeficiency Selected for Oral... - May 8th, 2022
- OPC UA for Sample Process Automation Improving Data Harmonization for Biobanks and Cell and Gene Therapies - Technology Networks - May 8th, 2022
- Alcyone Announces Two Oral Presentations on its Gene Therapy Platforms at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting -... - May 8th, 2022
- Decibel Therapeutics to Present at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting - GlobeNewswire - May 8th, 2022
- New treatment for infants with weakened immune systems - Sciworthy - May 8th, 2022
- Selecta Biosciences Announces Six Presentations at the Upcoming 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) - Yahoo... - May 8th, 2022
- CODA Biotherapeutics Announces Preclinical Data From Epilepsy Program to be Presented at American Society of Gene & Cell Therapy 25th Annual... - May 8th, 2022
- Gene Therapy Cell Culture Media Market Scope and overview, To Develop with Increased Global Emphasis on Industrialization 2029 Fujifilm Holdings... - May 8th, 2022
- Research Roundup: COVID-19 Cognitive Impairment Equal to 20 Years of Aging - BioSpace - May 8th, 2022
- International Thalassaemia Day 2022: The genetic perspective of Thalassemia - Free Press Journal - May 8th, 2022
- Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Tre - Benzinga - May 8th, 2022
- Virica Biotech Announces Collaboration with the Government of Canada - Business Wire - May 8th, 2022
- Tenaya Thearpeutics Reports Will Present Preclinical Data On Gene Therapy Programs, Platform Capabilities - Benzinga - May 8th, 2022
- Precision BioSciences to Present Preclinical In Vivo Gene Editing Research at Upcoming American Society of Gene & Cell Therapy 25th Annual Meeting... - May 8th, 2022
- Catalent introduces cryogenic capabilities to support growing demand for cell and gene therapy - gasworld - April 17th, 2021
- Bioprocessing and Bioproduction Trends in Cell and Gene Therapies - Technology Networks - April 17th, 2021
- Alliance for Cancer Gene Therapy Summit 2021 Features World Renowned Cancer Researchers Advancing Solid Tumor Breakthroughs - WFMZ Allentown - April 17th, 2021
- Gene therapy startup StrideBio signs collaboration deal with Duke University - WRAL Tech Wire - April 17th, 2021
- Cell and gene therapy services market forecast to see significant growth over the next decade - BioPharma-Reporter.com - April 17th, 2021
- TeamedOn and AGTC Announce a Licensing Agreement Advancing X-Linked Retinoschisis Gene Therapy Program - BioSpace - April 17th, 2021
- Jaguar Gene Therapy gains $139 million from investors - Daily Herald - April 17th, 2021
- Medically Necessary: Growth of biologics and gene therapies means more cold chain - FreightWaves - April 17th, 2021
- Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day - BioSpace - April 17th, 2021
- From gene therapies to medical treatments, cutting-edge research finds a home in NC - WRAL.com - April 17th, 2021