Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Chris Spivey

Brian Feth and Jonathan Grinstein go behind the headlines to discuss the impact of a Republican administration on Federal Trade Commission actions, tax relief, and Health and Human Services (HHS) leadership; as well as advancements in CAR-T.

Behind the Headlines: What the US Election Results Mean for Industry, In Vivo CAR-T Applications, and more

 is a bi-weekly panel discussion examining the latesttrends, readouts, and other factors that drive pharmaceutical news and innovation.Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seekingto highlight the more enduring lessons that hide behind the headlines.

In episode 6, Brian Feth, CEO atXcell Biosciences, and Jonathan Grinstein, North American Editor, 

, go behind the headlines to understand how a recently elected Republican administration in the U.S. might spur investment into the biopharmaceutical industry, particularly through tax breaks and loosening of certain regulations. They also discuss how stem cell research might be slowed and how nontraditional leadership of HHS might influence vaccine policy and pandemic preparedness.

FDA approval of Autolus Therapeutics' obecabtagene autoleucel

 (Aucatzyl) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) (1). This autologous CAR T-cell therapy, targeting CD19-positive B-cells, has shown promising results in the FELIX trial, with a notable complete remission rate. However, in line with FDA’s current warnings for treatment-related secondary cancer, “The approval includes a boxed warning for risks of cytokine release syndrome (CRS), immune effector cell–associated neurotoxicity syndrome (ICANS), and potential T-cell malignancies.”(1) The panel also noted Autolus’ other updates, including the first dosing of patients in their phase 1 CARLYSLE trial of obecabtagene autoleucel in refractory systemic lupus erythematosus (SLE)(2).

Sticking to CAR-T breakthroughs for B-cell abnormalities, the panel also pointed out that Interius BioTherapeutics dosed the first-ever patient with an in vivo CAR gene therapy for the treatment of B-cell malignancies. This is a possible game-changer for advanced therapies as in vivo therapies remove many of the lengthy and expensive manufacturing steps currently required for cell and gene therapies. Interius president and CEO, Phil Johnson, MD, said, “This milestone marks the first time that a durable in vivo CAR therapy has been used in the clinic. INT2104 has the potential to overcome ex vivo CAR therapy challenges with a single-dose, off-the-shelf, widely accessible therapy for the treatment of B cell malignancies enabled by Interius’ programmable platform.” (3)

1. FDA approves obecabtagene autoleucel for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. FDA. November 8, 2024. Accessed November 8, 2024. 

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-obecabtagene-autoleucel-adults-relapsed-or-refractory-b-cell-precursor-acute

2. Autolus Therapeutics reports third quarter 2024 financial results and business updates. Press release. Autolus Therapeutics. November 12, 2024. 

https://autolus.gcs-web.com/news-releases/news-release-details/autolus-therapeutics-reports-third-quarter-2024-financial

3. Interius BioTherapeutics doses first patient with in vivo chimeric antigen receptor (CAR) gene therapy for B-cell malignancies. Press release. Interius BioTherapeutics. October 23, 2024. 

https://interiusbio.com/2024/10/interius-biotherapeutics-doses-first-patient-with-in-vivo-chimeric-antigen-receptor-car-gene-therapy-for-b-cell-malignancies/

Videos

Behind the Headlines: What the U.S. Election Results Mean for Industry, In Vivo CAR-T Applications, and more 

Marianna Tcherpakov, PhD, director of Business Development, CMC Services, at Frontage Laboratories, spoke on the CDMO’s role in tackling complex technical challenges to enable startup biotechs solve scale up problems.

BIO 2024: CDMOs Focus on Complex Technical Challenges to Boost Startup Biotechs

Contract development and manufacturing organization (CDMO) Frontage Laboratories founder Song Li, PhD, who currently holds the position of executive chairman, board of directors, at the company, first set up a testing laboratory in Thorofare, NJ, with a focus on analytical and bioanalytical testing that quickly established a reputation for expertise. That foundation of expertise seems to have stayed fairly well intact, and science—and scientists—driving projects forward appear to be the key selling point for this CDMO, which says of itself, “our scientific knowledge base, technical expertise and reputation for high quality services have been integral to our ability in turning our services into your solutions” (1). In speaking with the company’s staff at BIO 2024, Pharmaceutical Technology Group’s Chris Spivey discerned that the practical upshot for the company has been that Frontage Laboratories often attracts the more vexing or technically challenging projects, and as a result, the company’s teams are now well situated to deal with the more difficult of perplexing projects for which others may not be as well prepared. As an example of the company’s preparedness, it states on the resources section of its website that it uses a next-generation sequencing (NGS)-based method, called the Frontage PGx NGS Test, to rapidly determine the genotype of 300+ genes involved in absorption, distribution, metabolism, and excretion (2).

Talking with Spivey at BIO, Marianna Tcherpakov, PhD, director of Business Development, CMC Services, Frontage Laboratories, at BIO, outlined the general approach the company takes when working with clients. She emphasized that the company’s approach is always customizable and personalized to not only the science the client requires but also to their specific strengths and weaknesses as an organization. “We look at your project as our own product. We therefore offer fast delivery, fast responses to client questions, almost as if we are holding hands … Many come from smaller startups that need a lot of guidance. [This] can be technical, it might be regulatory, but it all relies on our very strong scientific base,” Tcherpakov said. “We are passionate about technology,” she added.

Many of the PhD staff at Frontage Laboratories have come from other companies in the industry, and Tcherpakov pointed out that these past experiences and learnings are often brough to bear on the current project challenge. “We understand how stressful it can be for the smaller biotech, and even middle-sized [biotechs], when you have a lot of deadlines. You have clinical trials submissions coming up, and you need to be on top of everything, and fast,” she said.

Touting their scientific backbone Frontage Laboratories advertises itself as “more than a CRO”, in that it is also a “full global pharmaceutical development organization. Frontage Laboratories offers a comprehensive suite of drug discovery and development services including chemistry, DMPK [drug metabolism and pharmacokinetics], preclinical safety/toxicology, and clinical trial services with strategic and regulatory support to effectively bring new lead candidates through the IND [investigational new drug] stage to Phase II” (3).

BIO 2024 was held on June 3–6 in San Diego, Calif.

1. Frontage Laboratories. Services & Solutions. 

 (accessed August 21, 2024).
2. Frontage Laboratories. Resource Library. 

 (accessed August 21, 2024).
3. Frontage Laboratories. About Frontage. 

Marianna Tcherpakov, PhD, is the director of Business Development, CMC Services, at Frontage Laboratories. Image courtesy of Frontage Laboratories.

Leaders in pharmaceutical industry discuss the latest news making waves in the pharmaceutical industry. 

Behind the Headlines, Episode 5: GLP-1 Shortage, Nobel Prize, RNA Editing, and More

 is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In episode 5, panelists Laks Pernenkil, Principal Life Sciences at Deloitte; Brian Feth, CEO at Xcell Biosciences; and Alex Philippidis, Senior Business Editor at Genetic Engineering & Biotechnology News, go behind the headlines to understand why FDA felt it was time to remove 

 from the drug shortages list (1) and how Catalent’s sale of a small molecule manufacturing facility (and incidentally its own headquarters) to expanding European company Ardena might affect future drug availability (2).

The panel also discussed Nobel Prize awards being given in the 

 field (3) and what that might mean in terms of growth, given a series of false starts and hurdles including the shelving of a hepatitis C therapy due to fears that microRNA might not compete commercially with traditional therapeutic approaches (4). The Nobel Prize in physics awarded to recognize advancements in artificial intelligence (5) was likewise considered from the perspective of how AI will bolster drug discovery and drug formulation, as well as help optimize drug manufacturing. 

Lastly, panelists discussed the emerging potential for novel protein replacement therapies on the heels of Wave Life Sciences’ positive clinical data for RNA editing to combat AAT protein deficiency (6), and how temporary and reversible RNA-editing approaches may confer additional benefits that further support this novel approach in the marketplace.

FDA clarifies policies for compounders as national GLP-1 supply begins to stabilize. FDA Drug Safety and Availability. October 2, 2024. 

https://www.fda.gov/drugs/drug-safety-and-availability/fda-clarifies-policies-compounders-national-glp-1-supply-begins-stabilize

Catalent signs agreement to sell Somerset, NJ oral solids facility. Catalent. October 14, 2024. 

https://www.catalent.com/catalent-news/catalent-signs-agreement-to-sell-somerset-nj-oral-solids-facility/

The Nobel Prize in physiology or medicine, 2024. 

https://www.nobelprize.org/prizes/medicine/2024/press-release/

Sparmann A and Vogel J. RNA-based medicine: from molecular mechanisms to therapy. 

https://www.nobelprize.org/prizes/physics/2024/press-release/

Wave Life Sciences Announces First-Ever Therapeutic RNA Editing in Humans Achieved in RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency. Press release. Wave Life Sciences. October 16, 2024. 

https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-first-ever-therapeutic-rna-editing

Laks Pernenkil, Brian Feth, and Alex Philippidis go behind the headlines to discuss the impact of recent news, including FDA’s drug shortage list update, Nobel prize winners in microRNA and AI, and a big-potential research win for RNA editing. 

Behind the Headlines: GLP-1 Shortage, Nobel Prize, RNA Editing, and More

Experts in cell and gene therapy weigh in on hot topics in a quick-hitting buy, sell, hold format before expanding their thoughts on complex questions. 

Buy, Sell, Hold: Cell and Gene Therapy - Part 2

 is a rapid-fire expert panel covering a single topic, such as lipid nanoparticle delivery, supply chain resilience, 

 (CGT), or aseptic filling. Panelists first respond to quick-hit questions with a buy, sell, hold format before being allotted more time to share more considerate responses to complex topics.

While finance and investment perspectives flavor the discussion, science and pragmatism are the main ingredients driving these dynamic panel discussions.

“Episode 1: Cell and Gene Therapy” features the expertise of Fabian Gerlinghaus, co-founder and chief executive officer of Cellares; Dr. Claudia Zylberberg, board chair and co-founder of Arscience Biotherapeutics, and board chair and co-founder of Akron Biosciences; and Benjamin McLeod, director, Cell & Gene Therapy Segment, at NanoMosaic.

In the first 2 segments, the panelists share quick thoughts on CGT topics, including: The merits of focusing on orphan diseases; training new staff versus re-training existing staff; machine learning, artificial intelligence, and quantum computing; existing supply chain functioning; government investment into CGT; using traditional CDMOs for CGT production; facility retrofits for CGT; and CGTs as first-line treatment.

In the final segment, panelists had up to two minutes each to weigh the advantages and disadvantages of gene editing (CRISPR), gene augmentation, and gene-silencing approaches. Panelists also discussed their technology or engineering wish lists, as well as how to best to sync up regulations to current technology progress. Lastly, they exchanged ideas about the key missing piece(s) to lowering cost and improving accessibility.

Click here for more episodes of Buy, Sell, Hold

Fabian Gerlinghaus, Co-Founder and CEO, Cellares

Fabian Gerlinghaus is Co-Founder and Chief Executive Officer of Cellares. He is driven by a strong sense of purpose and is passionate about building the future of cell therapy manufacturing. With 10+ years of experience as an innovator and a leader, Fabian has established a track record of assembling top-performing teams to successfully drive novel bioprocessing technologies from ideation to commercial readiness. Prior to co-founding Cellares, Fabian served as Chief Innovation Officer at Synthego, where he co-invented the company’s proprietary RNA synthesizer technology and helped grow the company from five to more than 230 employees. He successfully led the interdisciplinary team that took synthesizer technology from whiteboard sketch to production-ready instruments within two years, enabling the company to be the first to market with its CRISPR/Cas9 product portfolio. He earned a master’s degree in aerospace engineering from the Technical University of Munich, and an honors degree in technology management from the Center for Digital Technology and Management, Munich

Dr. Claudia Zylberberg, Board Chair and Co-Founder Arscience Biotherapeutics, Board Chair and Co-Founder Akron Biosciences

With over 30 years of experience in the regenerative medicine industry, Dr. Zylberberg has established herself as a leader in her field. For nearly 15 years, she led Akron Bio as its founder and CEO with leadership, foresight, and expertise in advanced therapies manufacturing. Her passion for pushing the industry forward in the development and commercialization of safe novel therapies also led her to co-found the Standards Coordinating Body and serve as an active member of numerous industry boards and advisory committees. Claudia co-founded AssureImmune, an adult stem cell bank, and worked with Nabi Pharmaceuticals, specializing in human plasma-derived products. Her experience in product development and protein manufacturing was instrumental in the development of key materials to accelerate the regenerative medicine industry. She holds numerous patents and developed several patent-pending platform technologies in cryopreservation, novel formulations, and others. Claudia received her PhD from the University of Buenos Aires, where she studied Immunology, and conducted her doctoral research at the University of British Columbia. She also authored a children’s book called You’re Full of Genes to introduce the younger generation to genetics. Claudia is a member of the Forum on Regenerative Medicine at the National Academies of Sciences, Engineering, and Medicine.

Benjamin McLeod, Director, Cell & Gene Therapy Segment, NanoMosaic

Currently Benjamin helps many related researchers become more familiar with CGT concepts by translating complex processes into digestible bites on social media platforms such as LinkedIn and X. At OmniaBio he was product manager, collaborating with technical stakeholders to develop clear and actionable messaging around OmniaBio’s manufacturing services. At Virica Biotech he was Technical Lead, for a company whose viral sensitizer technology, increased viral yield in manufacturing, and potency in viral therapeutic products.

Cell and gene therapy experts Fabian Gerlinghaus, Dr. Claudia Zylberberg, and Benjamin McLeod weigh in on hot topics in CGT. 

Chris Spivey

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