Behind the Headlines Episode 27

Ask the Expert

Small Molecules, APIs, and Excipients—Trends, Challenges, and Opportunities

Drug Digest September 2025

Artificial intelligence, machine learning, and novel instruments are helping drug developers.

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

J.P. Morgan Report Lists Biologics, Obesity, China

Top Trends Reshaping Pharma R&D

AGC Biologics announced on Aug. 9, 2023 the completed expansion of its manufacturing space at the Milan Cell & Gene Center of Excellence production. The expansion offers the addition of 1 x 50L, 2 x 200L, and 2x 1000L suspension single-use bioreactors and allows for up to four iCellis500 adherent single-use bioreactors to meet demands for upcoming good manufacturing practice (GMP) viral vector manufacturing projects, according to the company press release.

Additionally, the project included the build-out of more process development lab space, a new automated filling line, and the addition of a new fully dedicated 1000-square-meter warehouse space.

“This is another important milestone for our site and for the AGC Biologics global Cell and Gene network as we prepare to serve more developers,” said Luca Alberici, General Manager, AGC Biologics Milan, in a press release. “Thanks to this expansion we have the flexibility to support companies from clinical to commercial stages for 

 gene therapy applications and can manufacture adeno-associated and lentiviral vectors from medium 50L scales to as large as 2000L. When you combine this with our development, analytical and fill/finish services, this site can offer cell and gene developers any type of support they need throughout their product’s lifecycle.”

The site was acquired by AGC Biologics in July 2020, and the Milan location is the first cell and gene therapy site approved in Europe for GMP manufacturing of clinical and commercial supplies, according to the press release. Further, the site has brought multiple products from the pre-clinical stage through commercial phases, such as developing a custom plug-and-play systems for vector manufacturing.

The two cell and gene facilities from AGC Biologics, in Milan and Longmont, US, receive support from the Biologics Heidelberg site, which produces proprietary plasmid starting materials for advanced therapies in high-quality and GMP grades, according to the press release.

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The site was acquired by AGC Biologics in July 2020, and the Milan location is the first cell and gene therapy site approved in Europe for GMP manufacturing of clinical and commercial supplies.

AGC Biologics Completes Expansion at CGT Manufacturing Site in Milan

Regeneron Pharmaceuticals and Decibel Therapeutics announced a definitive agreement on August 9, 2023, for the acquisition of Decibel by Regeneron at $4 per share of Decibel common stock payable in cash at closing.

“We at Decibel are deeply committed to discovering and advancing innovative new therapies with the potential to be transformative for people with severe forms of hearing loss. We have built a pipeline of gene therapy product candidates for the ear that we believe have such potential. After full consideration, the Decibel Board has determined that this transaction is the best way to maximize shareholder value and ultimately benefit patients,” said Laurence E. Reid, PhD, president and chief executive officer of Decibel, in a press release. “We have collaborated with our colleagues at Regeneron for the past six years and have huge respect for their research and development capabilities. We have full confidence that with Regeneron’s expertise and resources the Decibel pipeline can be optimally developed, and our team is committed to enabling that long-term success.”

The initial collaboration between the two companies began in 2017 with an extension announced in 2021, and they are now developing three gene therapy programs targeting different forms of congenital, monogenic hearing loss, according to the press release. Decibel’s lead investigational candidate, DB-OTO, is currently in the global Phase I/II CHORDTM clinical trial, which is an investigational cell-selective, adeno-associated virus gene therapy. The objective of the study is to provide physiological hearing to individuals with congenital hearing loss caused by mutations of the otoferlin gene.

“We are delighted to announce the planned acquisition of Decibel, who have been long-standing collaborators, notable for their deep scientific knowledge and commitment to people with hearing loss,” said George D. Yancopoulos, MD, PhD, Board co-Chair, chief scientific officer and president of Regeneron, in a press release. “DB-OTO, our shared lead investigational gene therapy, will soon reach patients in its first clinical trial, offering new promise to children with this rare form of congenital hearing loss, as well as potential proof-of-concept for future gene therapies addressing more common forms of genetic hearing loss. We believe that Decibel’s assets and specialized team will further strengthen our genetic medicines portfolio, enabling Regeneron to accelerate the development of innovative genetic therapies and a rich pipeline of hearing loss treatments.”

The transaction is expected to close in the third quarter of 2023, according to the press release.

The initial collaboration between the two companies began in 2017 with an extension announced in 2021.

Regeneron Pharmaceuticals to Acquire Decibel Therapeutics 

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

Highlights from a wide ranging discussion on COVID-19 pandemic response, future government communications, and healthcare funding for mRNA with experts Dr. Kate Broderick, Chief Innovation Officer, Maravai LifeSciences, and Dr. Tom Madden, President & CEO, Acuitas Therapeutics.

Topics include supply chain modalities, ensuring a fully trained manufacturing workforce, combining delivery with gene editing innovations, epigenetic approaches to modulate gene expression moving toward the clinic. We conclude with the huge potential for RNA expression of monoclonal antibodies and protein replacement applications.

Catch the full podcast episode featuring Broderick and Madden when it goes live at the 

Dr. Kate Broderick is Chief Innovation Officer at at TriLink Biotechnologies, a Maravai LifeSciences company. Prior to joining TriLink, Kate spent 15 years with Inovio Pharmaceuticals where she assumed positions of increasing responsibility, starting as Scientist, to her most recent position as Senior Vice President, R&D. She received her B.Sc. in Genetics and her Ph.D. in Molecular Genetics from the University of Glasgow.

Dr. Tom Madden is President & CEO at Acuitas Therapeutics. A world-renowned expert in the area of nanotechnology. Dr. Madden co-founded Acuitas Therapeutics in February 2009 and has guided the company into its position as a global leader through the development and application of lipid nanoparticle (LNP) technology. Dr. Madden obtained his BSc. and Ph.D. in Biochemistry from the University of London, U.K.

Highlights from a wide ranging discussion on COVID-19 pandemic response from Kate Broderick, Chief Innovation Officer at Maravai LifeSciences, and Tom Madden, President & CEO at Acuitas Therapeutics.

Unpacking COVID-19 Pandemic Response

Sparian Biosciences announced on Aug. 8, 2023 that it has been awarded a $19 million, five-year grant to fund development of SBS-518 from investigational new drug (IND) submission through Phase I clinical development.

According to the press release, SBS-518 is a first-in-class dual sigma receptor (SR) antagonist/dopamine transporter (DAT) inhibitor in development for the treatment of stimulant use disorder (StUD). Further, StUD is a subset of substance use disorders, including cocaine and methamphetamine use disorders.

In pre-clinical efficacy assays, SBS-518 blocks the stimulant induced rise in extra-cellular dopamine and blocks cocaine and methamphetamine self-administration without itself producing self-rewarding behavior, according to the press release. Additionally, dopamine active transporters, or DAT, have been proven to be an indefinable target for therapeutics.

“Sparian was founded to develop innovative therapeutics to treat CNS disorders, particularly pain and substance use disorders,” said Jeff Reich, MD, Chief Executive Officer and Co-founder, in a press release. “StUD presents a major unmet medical and public health need for which there are currently no approved therapies. If we can successfully advance SBS-518 into the clinic, it could represent a significant step forward in the treatment of substance use disorders.”

This grant is the third NIH/NIDA grant that Sparian has received, and the company has been awarded a total of $41 million in government grants to support 3 programs. It was awarded by NIH/NIDA under the Help End Addiction Long-Term (HEAL) initiative. Its lead program, SBS1000, is now in Phase 1 clinical trials, and the dual mu opioid receptor, SBS-226, is in development for opioid use disorder (OUD).

“We are extremely proud of the ongoing support we have received from NIDA,” Reich said in a press release. “NIDA, through the HEAL initiative, has sparked a new wave of innovation and entrepreneurship dedicated to the treatment of substance use disorders. Sparian, along with our academic partners, is honored to be a trusted collaborator.”

SBS-518 is a first-in-class dual sigma receptor (SR) antagonist/dopamine transporter (DAT) inhibitor in development for the treatment of stimulant use disorder (StUD).

Sparian Biosciences Awarded Five-Year NIH/NDA Grant for Development of SBS-518

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In a press release on Aug. 8, 2023, Lisata Therapeutics announced FDA’s Orphan Drug Designation had been granted to LSTA1 for the treatment of malignant glioma. Lisata Therapeutics is a clinical-stage pharmaceutical company developing therapies for advanced solid tumors and other major diseases. The company created LSTA1 to activate a new uptake pathway, allowing co-administered or tethered antineoplastic drugs to combat solid tumors more successfully. The drug can also modify the tumor microenvironment to make tumors more vulnerable to immunotherapies.

LSTA1 is currently undergoing multiple global studies in a range of solid tumor types in combination with a range of antineoplastic regimens. In the near future, Lisata plans to collaborate with researchers from the University of Tartu in Estonia to conduct a clinical study evaluating LSTA1 in previously untreated glioblastoma multiform (GBM). The company plans to study the impact of LSTA1 when added to standard-of-care (SoC) to treat GBM. The study will be conducted at multiple sites in Estonia and Latvia, with an estimate of 30 patients (with a randomization of 2:1 LSTA1 + SoC vs placebo + SoC). Lisata expects the first patient will be treated in the fourth quarter of 2023.

“Malignant glioma is one of the most aggressive and deadly malignancies,” said Kristen K. Buck, M.D., executive vice president of R&D and chief medical officer of Lisata, in a company press release. “This Orphan Drug Designation acknowledges the high unmet medical need of this patient population as well as the potential of LSTA1 to benefit patients in this setting.”

The Orphan Drug Designation is given by FDA to drugs or biologics designed to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States. These designations create fiscal incentives for clinical development and a seven-year period of US market exclusivity if FDA approval is met. A large amount of non-clinical data suggests that LSTA1 enhances a range of antineoplastic therapies; LSTA1 also showed positive safety, tolerability, and activity in clinical trials enhancing standard-of-care chemotherapy for pancreatic cancer. Lisata anticipates LSTA1 will assist with a range of treatments to treat solid tumors more effectively.

The investigational drug was developed to activate a novel uptake pathway, allowing antineoplastic drugs to combat solid tumors more successfully.

FDA Grants Orphan Drug Designation to Treatment of Malignant Glioma 

Emerson announced on August 3, 2023 that it is collaborating with Sartorius to integrate Sartorius’s Biostat STR Generation 3 family of bioreactors with Emerson’s DeltaV distributed control system (DCS). According to the company press release, the Biostat STR Generation 3 for Emerson’s DeltaV, a variant of the Biostat STR Generation 3 family, is a pre-engineered solution that delivers intuitive connectivity for accelerating the process of bringing therapies to market.

“One of the biggest barriers to fast delivery of new treatments is the time it takes to connect new equipment to the plant’s control system,” said Nathan Pettus, president of Emerson’s process systems and solutions business, in a press release. “Working closely with Sartorius to seamlessly incorporate our DeltaV technologies and software into Sartorius bioreactors, we jointly have made one of the most critical components of biopharmaceutical manufacturing ready to connect out of the box for faster speed-to-market.”

The bioreactors can integrate with Emerson’s DeltaV PK Controller in addition to S-Series and M-Series controllers. Further, built-in templates can provide standardized configurations for the most common uses, which allow teams to use standard setups to bring operations to a safer, quicker shift for market needs.

“The biopharmaceutical industry requires modular and scalable bioprocess equipment to help quickly and easily manufacture the treatments patients around the world are waiting for,” said Mario Becker, head of bioreactor technologies at Sartorius, in a press release. “By integrating Emerson software and technology into our new Biostat STR DeltaV variant, we help our customers rapidly bring their operations online while also maintaining flexibility, scalability, and the current Good Manufacturing Practice strategies that are critical to delivering high-quality treatments.”

The goal of the easy configuration and integration of the Biostat STR Generation 3 bioreactors is intended to help manufacturers build a connected plant. Additionally, increased access to advanced automation strategies and diagnostics from the DeltaV DCS help manufacturing plants, as well.

The bioreactors can integrate with Emerson’s DeltaV PK Controller in addition to S-Series and M-Series controllers.

Sartorius and Emerson Collaborate to Expedite New Therapies to Market

- Current state of the cellular therapy market and challenges bringing newer therapies to commercialization

- Critical components tied to successful drug development and manufacturing of cell therapies

- Remaining current with regulatory guidance tied to cell therapies

- Proper oversight of supply chains tied to a cell therapy project

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Catalent's Martin De Brauwere and Maria Lopez react to cell therapy innovations and highlight strategies to optimize the drug development and manufacturing process with assistance from a CDMO.

Solutions to Optimize Cell Therapy Development and Manufacturing

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On August 1, 2023, FDA warned consumers that two lots of Tydemy (drospirenone/ethinyl estradiol/levomefolate calcium), a prescription oral contraceptive manufactured by Lupin Pharmaceuticals, could be less effective because of lower levels of ascorbic acid, an active ingredient, in the product. These lots (L200183 and L201560) were dispensed in the United States from June 3, 2022, to May 31, 2023. The reduced effectiveness could result in unexpected pregnancies. FDA wrote that as of August 1, 2023, FDA “has not received any reports of adverse events related to using this product.”

On July 29, 2023, Lupin warned its consumers about its 

voluntary recall of the two lots of Tydemy

. Lupin is notifying its wholesalers, drug chains, distributors, supermarkets, and mail order pharmacies via phone and recall notification to arrange the return of all recalled product lots. The company advised patients to keep taking their medication, but to immediately contact their health care professional about alternative contraceptive methods. Wholesalers, distributors, and retailers that have the recalled Tydemy should stop distribution of the recalled product immediately. Lupin had formerly voluntarily recalled the two lots of Tydemy at the wholesale level.

FDA wrote that it encourages healthcare professionals and patients to report any adverse events or quality issues with these or any medications to FDA’s 

Lower levels of an active ingredient may result in unexpected pregnancies. 

FDA Warns that Tydemy May Have Reduced Effectiveness

BlueRock Therapeutics and bit.bio announced a collaboration and option agreement for the discovery and manufacture of iPSC-derived regulatory T cells for use in creating therapeutics on August 3, 2023, according to a company press release.

“Tregs play a crucial role in maintaining balance in the body’s immune system and controlling excessive immune reactions,” said Stefan Irion, MD, Chief Scientific Officer of BlueRock Therapeutics, in a press release. “iPSC-derived Treg based therapies have the potential to treat a broad range of autoimmune and inflammatory disorders, and we look forward to collaborating with the bit.bio team to explore how their opti-ox cell programming technology can accelerate our efforts to discover and manufacture Tregs from iPSCs.”

With the new agreement, bit.bio will use its machine learning platform to identify transcription factor (TF) combinations for reprogramming iPSCs into Tregs, according to the press release. Further, the new agreement includes different alternatives for BlueRock to license bit.bio’s opti-ox precision cell programming technology to control the expression of TF combinations within Treg cell therapies. The opti-ox uses a dual genomic safe harbor approach to cell programming, and bit.bio uses opti-ox to drive the rapid TF-mediated conversion of iPSCs into highly defined cell types in a single step, according to the company.

“We are delighted to partner with the team at BlueRock, who are world-leaders in iPSC-derived cell therapies, and together address the significant unmet needs of these patients,” said Mark Kotter, MD PhD, CEO of bit.bio, in a press release. “Today’s announcement marks an important milestone for bit.bio. As well as providing significant financial contributions, our collaboration is a testament to the unique capabilities of bit.bio’s team.”

In addition, BlueRock will be responsible for the global development and commercialization of therapeutic candidates emerging from this collaboration, according to the press release.

BlueRock will be responsible for the global development and commercialization of therapeutic candidates emerging from this collaboration.

BlueRock Therapeutics and bit.bio Collaborate for Manufacture of Regulatory T cell Based Therapies

On Aug. 1, 2023, US-based iTolerance, an early-stage regenerative medicine company, and Lithuania-based Northway Biotech, a contract development and manufacturing organization, announced that they have entered into an agreement for the manufacturing of Streptavidin-FasL (SA-FasL) fusion protein, an important component of iTolerance’s proprietary iTOL-100 platform technology.

According to a company press release, the SA-FasL fusion protein has demonstrated promising potential in pre-clinical studies; it was shown to establish durable, localized immune tolerance, which would thus enable implanted tissue or organoid, or a cell therapy, to function as a viable replacement for damaged native cells.

Under the agreement, Northway Biotech will use its expertise to develop a robust manufacturing process for SA-FasL. Process development work will involve the production of a master cell bank, the development and qualification of appropriate analytical methods, the optimization of formulation, and the development and scaling up of the production process. Northway Biotech will also be responsible for manufacturing a current good manufacturing practice-produced batch of drug substance for clinical studies.

“We believe this strategic partnership is not only a significant step for iTolerance, but also in developing advanced regenerative medicine technologies. We are incredibly delighted to establish this important partnership with Northway Biotech … to manufacture the clinical supplies necessary for our first in human clinical study for our lead program, iTOL-102,” said Anthony Japour, CEO of iTolerance, in the press release. “We look forward to working closely with the Northway Biotech team to advance our iTOL-100 technology and progress towards providing treatment options that have the potential to cure diseases without the need for chronic immunosuppression.”

“We believe this partnership is an exciting development in the regenerative medicine field, and we are honored to play a role in advancing this important work. With a commitment to meeting contractual timelines, our primary objective is to ensure the punctual delivery of these crucial materials to our esteemed partners at iTolerance,” said Vladas Algirdas Bumelis, CEO and chairman of the board at Northway Biotech, in the release.

Under a manufacturing agreement, Northway Biotech will develop a manufacturing process for iTolerance’s fusion protein in development for regenerative therapy.

Featured Content

Behind the Headlines Episode 27

Ask the Expert

Drug Digest September 2025

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

Top Trends Reshaping Pharma R&D