Feliza Mirasol is the science editor for 

Feliza Mirasol

Scinai Immunotherapeutics (Scinai), an Israel-based biopharmaceutical company focused on developing inflammation and immunology biological products, announced on Dec. 16, 2024 the establishment of a US-based subsidiary for its contract development and manufacturing organization (CDMO) services. The company provides CDMO services through its Scinai Bioservices business unit. This US subsidiary, incorporated in Delaware, will operate under the name Scinai Bioservices Inc.

The Scinai Bioservices CDMO business unit was launched in 2024 and operates from the company's site in Jerusalem, Israel. The Jerusalem facility comprises 1850 square meters (approximately 20,000 square feet) of clean rooms and laboratories, which operate under current good manufacturing practice (CGMP) conditions in compliance with European Medicines Agency (EMA) and FDA requirements, according to a company press release (1).

"We are thrilled and energized by the opening of our new subsidiary in the [United States]. Last week, we signed our first contract with US customer, Serpin Pharma, to support their clinical manufacturing,” said Amir Reichman, CEO of Scinai, in the press release (1). “I believe that our expanded presence in the US will significantly enhance the prospects of our CDMO unit."

From its launch, the CDMO unit has executed drug development projects for nine different biotech companies, which included upstream and downstream process development, scale-up, analytical methods development, and drug manufacturing for pre-clinical and clinical supplies. The company has also partnered with Ayana Pharma, an Israel-based pharmaceutical company specializing in liposomal therapeutics for treating cancer, under which Scinai will provide liposomal encapsulated drug development services.

The Scinai Bioservices unit operates on projects at the early stage, where the company noted in its press release capacity is strained because of the complex manufacturing processes associated with biologics. “Additionally, the BIOSECURE Act, introduced in January 2024, aims to prevent ‘foreign adversary biotech companies’ from receiving US federal funding,” the company stated in its press release (1).

Scinai's new US subsidiary aims to serve US-based early stage biotech companies with the goal of providing required CDMO services that are currently in short supply. The US subsidiary will also allow early stage biotech startups, which are supported by US government grant funding, to use their grant money in contract with Scinai, which would allow them to avoid concerns related to the BIOSECURE Act, Scinai stated in its press release.

To put the situation into context, Parviz Shamlou, senior vice-president of Science and Technology, Abzena, spoke to the PharmTech Group during BIO 2024 this past June about the impact the act will have on the bio/pharmaceutical industry and how the industry is preparing for it. “We appreciate that [the BIOSECURE Act] is an important act designed to protect the health of citizens of this country. And we know we must do everything we can to avoid any unintended, potentially harmful use of our services as highlighted in the act. [We] have put in place a robust contingency plan as well as a CMC [chemistry, manufacturing, and controls] strategy that are designed to mitigate any such risk to supply chain and the development of the assets that our clients bring to us,” Shamlou stated in the interview (2).

1. Scinai Immunotherapeutics. Scinai Immunotherapeutics Establishes US Subsidiary for CDMO Business Unit, Scinai Bioservices Inc. Press Release. Dec. 16, 2024.
2. Haigney S. 

Impact of Biosecure Act on the Pharma Industry (BIO 2024)

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Scinai Bioservices Inc. has been established in Delaware as the company's new US-based subsidiary, which will serve biotech companies in early stage drug development.

Scinai Immunotherapeutics Establishes US CDMO Business Unit

AbbVie has signed a definitive agreement to acquire Nimble Therapeutics, a biotechnology company specializing in oral peptide therapeutics, which would add Nimble’s lead asset, an investigational oral peptide interleukin 23 receptor (IL23R) inhibitor that is in preclinical development for treating psoriasis. to AbbVie's immunology pipeline. In addition to the lead asset, AbbVie would also acquire a pipeline of other novel oral peptide candidates that show potential across several autoimmune diseases, AbbVie announced in a Dec. 13, 2024 company press release.

With the acquisition, AbbVie also gains Nimble's peptide synthesis, screening, and optimization platform. This platform uses proprietary technology to facilitate rapid discovery and optimization of peptide candidates for a range of targets.

Under the agreement, AbbVie will pay $200 million in cash at the close of transaction, which is subject to certain customary adjustments. AbbVie will also pay certain interim funding payments, and Nimble's shareholders will be eligible to receive potential payment upon the achievement of a development milestone, according to the press release (1).

"The addition of Nimble's pipeline to AbbVie's existing pipeline, combined with our deep clinical and translational expertise in immunology, represents an important growth opportunity," said Jonathon Sedgwick, PhD, senior vice-president and global head of discovery research, AbbVie, in the press release (1). "Together, AbbVie and Nimble have the potential to help address the significant unmet medical need for people living with autoimmune diseases."

Nimble's lead candidate, which targets IL23R, has potential in the treatment of psoriasis and inflammatory bowel disease (IBD). According to AbbVie, IL23R is a clinically validated therapeutic target in certain autoimmune diseases and is known to be a major contributing factor in psoriasis and IBD pathogenesis as well as progression by way of increased inflammation and amplified immune responses (1).

"Nimble Therapeutics is committed to transforming the discovery of oral peptide-based medicines. With AbbVie's … expertise in developing and commercializing medicines on a global scale, Nimble's novel oral therapies will be well-positioned to reach more people living with autoimmune diseases," said Jigar Patel, PhD, founder and chief executive officer, Nimble Therapeutics, in the release. "The talented, passionate, and dedicated team at Nimble has made great progress over the past few years, and we are pleased that AbbVie has recognized the tremendous potential of our proprietary platform and emerging immunology pipeline."

Nimble is the latest in a series of acquisitions that AbbVie has conducted this year. Also in December 2024, AbbVie completed its 

$1.4 billion acquisition of Aliada Therapeutics

$8.7 billion acquisition of Cerevel Therapeutics

, both of which strengthen AbbVie’s neuroscience pipeline (2,3). In June 2024, AbbVie 

acquired Celsius Therapeutics for $250 million in cash

, which included an antibody drug candidate in clinical development for IBD (4). In May 2024, AbbVie completed its acquisition of Landos Biopharma for approximately $137.5 million in cash, which included that company’s lead asset, NX-13, an oral nucleotide oligomerization domain-like receptor family member X1 agonist in clinical development for treating ulcerative colitis and Crohn's disease (5).

1. AbbVie. AbbVie to Acquire Nimble Therapeutics, Further Strengthening Immunology Pipeline. Press Release. Dec. 13, 2024.
2. AbbVie. AbbVie Completes Acquisition of Aliada Therapeutics. Press Release. Dec. 11, 2024.
3. AbbVie. AbbVie Completes Acquisition of Cerevel Therapeutics. Press Release. Aug. 1, 2024.
4. AbbVie. AbbVie Acquires Celsius Therapeutics. Press Release. June 27, 2024.
5. AbbVie. AbbVie Completes Acquisition of Landos Biopharma. Press Release. May 23, 2024.

AbbVie's latest acquisition target is Nimble Therapeutics, which will give AbbVie a lead asset, an oral peptide for treating psoriasis.

AbbVie to Boost Immunology Pipeline with Acquisition of Nimble Therapeutics

Part two Colin McKinlay Nutcracker Therapeutic trends future mRNA LNP development.

Talking mRNA–LNP Formulation Trends (Part Two)

One of the exciting things in messenger RNA (mRNA) development is the fact that not everyone involved in this field is necessarily going in the same direction, says Colin McKinlay, PhD, senior director, Chemistry and Delivery Technologies, Nutcracker Therapeutics, in part two of this video interview. “There [are many] different places that mRNA can be applied, and, in some cases, moving past the early stages of this field, one of the things that people are going to have to do is start specializing in different areas,” he says.

“Vaccines is obviously an area that mRNA has really demonstrated itself already, and so, I think making sure that the platform technology doesn't get pigeonholed into only being something that people think of as a vaccine technology is … important for the growth of the field, and moving beyond infectious disease vaccines, or even COVID[-19] vaccines,” McKinlay explains.

“In terms of the LNP [lipid nanoparticle] side of things, where I spend [much] of my time, I think there [are many] lipid nanoparticle technologies that will focus on expression in the liver and are directed towards the liver. One of the things that [many] folks in the field are really focusing on is, how do we actually direct these lipid nanoparticles to other organs in the body so that we can address other types of disease areas? Those are things where there hasn't been … [much] large-scale clinical successes,” McKinlay adds.

LNPs for RNA delivery, in terms of non-viral RNA delivery, have dominated the field in terms of clinical approvals, but McKinlay expresses concern about the industry possibly getting stuck in a kind of local maxima, “where the field does [much] optimization and [may] climb in efficacy, but then … reach a point where there's not too much that [one] can really optimize on anymore. [At Nutcracker Therapeutics], we're able to branch away from traditional LNP type particles, and have particles that are a little bit more unique to the chemistry of … peptoid molecules, and we think that that's pushing us over a little bit away from where the rest of the field is working, so that there's maybe a little bit more room to grow and a little bit more room to improve,” McKinlay states.

Click above for part two of this video interview. Click 

Colin McKinlay, PhD, Senior Director, Chemistry and Delivery Technologies, Nutcracker Therapeutics

Colin McKinlay is the senior director of Chemistry and Delivery Technologies at Nutcracker Therapeutics. He has over 10 years of expertise in nucleic acid delivery through his current position at Nutcracker Therapeutics and his doctoral research at Stanford University with Professors Paul Wender and Robert Waymouth. This research involved preparation of synthetic delivery vehicles through organocatalytic ring-opening polymerization, and their characterization and 

evaluation for the delivery of DNA, RNA, and other biological macromolecules. He holds over five patents and nine peer-reviewed publications on chemical delivery technologies. McKinlay has led the delivery efforts at Nutcracker Therapeutics since 2018, including the establishment of its unique peptoid-based Nutshell delivery platform.

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In the second part of a video interview, Colin McKinlay, PhD, senior director, Chemistry and Delivery Technologies, at Nutcracker Therapeutics, discusses trends and the future direction of mRNA–LNP development.

Colin McKinlay, the senior director of Chemistry and Delivery Technologies at Nutcracker Therapeutics, discusses current challenges in mRNA and LNP manufacturing as well as innovations that meet these challenges.

Addressing Challenges in mRNA and LNP Manufacturing (Part One)

Two of the challenges that Nutcracker Therapeutics, a US-based RNA medicines company, has been working through are how to approach the manufacturing of RNA-based drugs for personalized therapies and how to bridge the gap between smaller-scale companies and clinical trial-scale studies, says Colin McKinlay, senior director, Chemistry and Delivery Technologies, Nutcracker Therapeutics.

“I think one of the lessons learned from the whole COVID[-19] pandemic and putting together the mRNA [messenger RNA]-based vaccines for COVID[-19] was that we could actually scale these up to … worldwide scales, and that we were able to do that fairly effectively. But starting to think about distributing and manufacturing mRNA drugs, where every patient has a different type of formulation, I think that starts to be a different type of challenge,” McKinlay says.

“[A]nother area that has [many] challenges associated with it is really being able to bridge smaller-scale companies … bridge their research work into Phase I, Phase II clinical-trial scale and get them materials that they need for conducting those trials,” he adds, noting that while there are many commercial suppliers available for research-grade mRNA and making mRNA at that small scale for drug discovery work, the cost for scaling up to larger-scale commercial manufacturing can be prohibitive. Therefore, there is need in the market for enabling small startups to scale up manufacturing, for both mRNA and lipid nanoparticles (LNPs).

“[Many] of the commercial options for making LNPs aren't really tailored towards … small scale. There [are] systems that are available from various … suppliers, but they generally have a pretty large minimum volume or minimum scale that they can operate at. So, what [many] people end up doing is resulting to just pipette-mixing or hand-mixing their components, which means that the LNPs [being made] are very different than the LNPs that [are] going to be [made] farther down in [the] development process and in … scale up,” McKinlay explains.

Click above for part one of this video interview.

Colin McKinlay is the senior director of Chemistry and Delivery Technologies at Nutcracker Therapeutics. He has more than 10 years of expertise in nucleic acid delivery through his current position at Nutcracker Therapeutics and his doctoral research at Stanford University with Professors Paul Wender and Robert Waymouth. This research involved preparation of synthetic delivery vehicles through organocatalytic ring-opening polymerization, and their characterization and 

 evaluation for the delivery of DNA, RNA, and other biological macromolecules. He holds over five patents and nine peer-reviewed publications on chemical delivery technologies. McKinlay has led the delivery efforts at Nutcracker Therapeutics since 2018, including the establishment of its unique peptoid-based Nutshell delivery platform.

On Dec 11, 2024, Intertek, a total quality assurance provider, announced that it entered into a partnership with CrystecPharma, a UK-headquartered crystal and particle engineering company that applies proprietary modified supercritical fluid (SCF) technologies to improve drug performance. The collaboration will aim to advance formulation science and accelerate development timelines for inhaled medicines, particularly dry powder inhaler (DPI) products.

Under the agreement, the companies will develop a “fast to clinic” platform that supports pharmaceutical clients by providing rapid DPI development—from the initial formulation concept to good manufacturing practice (GMP) clinical manufacturing. The companies are aiming to accelerate DPI development to less than half the time typically required. The agile, rapid framework will be designed to allow clients, who are looking to formulate small or large (biologics) molecules, or who are looking to repurpose existing APIs, to benefit from technology that enables crystallization of new forms of molecules. Clients will also have access to technology that allows for the manufacture of novel particles in ways that significantly enhance those particles’ therapeutic performance, according to a company press release.

“Combining the proprietary mSAS [modified supercritical anti-solvent] technology and the speed of early development that partnering with CrystecPharma affords, with the GMP manufacturing and analytics expertise at Intertek, provides the market with an unparalleled service in this area. This unique platform will enable highly effective, stable, and safe DPI medicines to be developed and brought to market in a fraction of the time previously possible,” said Chris Vernall, commercial director, Intertek, in the press release (1).

CrystecPharma’s proprietary drug formulation technology, mSAS, aims to improve the performance of therapeutics by using a supercritical fluid—typically carbon dioxide because of its low critical point conditions and attractive physical properties—to remove solvent rapidly from a drug solution. This rapid solvent removal allows the drug to precipitate as dry particles. SAS has many advantages, particularly its ability to crystallize drug molecules and remove solvent in a way that only parts per million is left behind in the final product. However, conventional approaches to using SAS have limitations. For instance, these processes are often unstable, difficult to scale, and the nozzles used are prone to blockage, according to CrystecPharma (2).

CrystecPharma’s technique allows many molecules that are traditionally challenging to formulate as powders to be developed in that format. The company also has a unique set-up that enables formulation and process development to occur in a matter of weeks, according to the company press release (1).

“The strength of our award-winning mSAS technology and Intertek’s expertise in analytical development and GMP clinical manufacturing creates unique opportunities to develop innovative, high-performing medicines and improved treatments for patients,” said Paul Thorning, co-founder and CEO of CrystecPharma, in the release (1).

The platform that Intertek and CrystecPharma are collaborating on will be made available starting in January 2025.

DPIs were initially developed for treating asthma and chronic obstructive pulmonary disease by delivering the therapeutic directly to the lungs. This direct-to-tissue delivery enables rapid action and lower doses than more conventional oral administration. In addition to other respiratory diseases, researchers are now exploring DPIs for other applications, including treatment of bacterial lung infections, delivery of vaccines, and systemic drugs such as insulin.

1. Intertek. Intertek Partners with CrystecPharma to Advance Formulation Science and Accelerate Development Timelines for Dry Powder Inhaler Products. Press Release. Dec. 11, 2024.
2. CrystecPharma. mSAS Technology. 

The partners will aim to establish a platform that enables rapid development of DPI products.

Intertek Teams Up with CrystecPharma on Formulation and Development of Dry Powder Inhaler Products

In December 2024, GSK entered into strategic deals with two companies with the goal of advancing drug development. GSK and Relation, a UK-based platform biotechnology company developing medicines for diseases with significant unmet need, formed two multi-program collaborations focusing on the identification and validation of novel therapeutic targets for fibrotic diseases and osteoarthritis. In a separate collaboration, GSK and Muna Therapeutics, a Denmark-based biotechnology company focused on developing innovative therapeutics for neurodegenerative diseases, will identify and validate novel drug targets for the treatment of Alzheimer’s disease.

Under the two collaboration agreements with Relation, GSK will pay a total upfront payment of $45 million, comprising an equity investment of $15 million, according to a Dec. 10, 2024 press release (1). Relation will be eligible to receive success-based collaboration payments of up to $63 million. It will also be eligible to receive potential preclinical, development, commercial, and sales milestone payments averaging $200 million per target across both deals as well as tiered royalties on net sales of products.

Fibrotic diseases and osteoarthritis are debilitating diseases that affect millions of patients worldwide. They represent areas of significant unmet medical need, according to Relation’s press release.

“These diseases have few treatments that address their root causes. By combining Relation’s patient-centric discovery platform with GSK’s global scale and expertise, we have the potential to accelerate the development of transformative medicines for patients who have long awaited new therapies,” said David Roblin, CEO of Relation, in the release (1). “Our approach of generating data directly from patient tissue allows us to build a comprehensive picture of the underlying human disease biology at scale. This, combined with our deployment of machine learning, enables us to make unprecedented inferences about how to effectively intervene in disease processes.”

In the collaboration with GSK, Relation will lead observational studies with the aim of generating two unique functional disease data sets. Meanwhile, Relation’s Lab-in-the-Loop platform will be used to analyze these data by integrating human genetics, single-cell multi-omics directly from human tissue, functional assays, and machine learning to discover novel disease targets. The company stated in its release that this approach minimizes the risk of clinical failure by ensuring that targets are robustly validated before entering the clinic. Relation will apply its platform to a number of related fibrotic conditions as well as osteoarthritis. GSK will have worldwide development and commercialization rights to any targets resulting from Relation’s studies.

“Working with Relation enables us to gain deep and precise insights into human causal biology and apply these to therapeutic discovery through a data-driven approach, supported by Relation’s proprietary platform,” said Kaivan Khavandi, senior vice-president, global head of Respiratory/Immunology R&D at GSK, in Relation’s press release (1). “The resulting targets will be supported by robust translational packages, which will in turn provide clarity on deployment in early clinical evaluation, ultimately supporting our broader portfolio ambition to develop new medicines for fibrotic diseases and osteoarthritis—where there remains significant unmet medical need.”

Under the deal with Muna Therapeutics, GSK will pay €33.5 million (US$35 million) upfront, and Muna will be eligible to receive up to €140 million (US$147 million) per target in milestone payments, according to a Dec. 5, 2024 press release (2). Muna will also be eligible to receive tiered royalties on net sales of products. GSK and Muna will explore insights from Muna’s MiND-MAP platform, a technology that applies spatial transcriptomics to brain samples from Alzheimer’s disease patients, cognitively resilient individuals, healthy controls, and centenarians with and without cognitive impairment. The platform generates a unique dataset of significant breadth and resolution that is expected to drive the discovery and development of innovative medicines for Alzheimer's disease, Muna stated in its company press release (2). GSK, in return, will lead drug development and take responsibility for preclinical activities, clinical development, manufacturing, and commercialization.

This collaboration leverages Muna’s expertise in mapping the brain’s response to pathological protein aggregates and the company’s all-in-human platform that identifies cellular mechanisms, gene networks, and molecular interactions underlying brain resilience. Any resulting candidate drug targets will be validated using Muna’s suite of humanized cell and animal models, which are supported by insights from patient tissue and biofluid samples.

“Our agreement marks a pivotal moment in Muna’s evolution and in the broader Alzheimer's research landscape. By combining GSK's commitment to breakthrough science with our MiND-MAP platform's ability to deliver novel insights into brain resilience, we aim to transform the landscape of drug discovery for neurodegenerative diseases and bring new hope to millions of patients worldwide,” said Rita Balice-Gordon, PhD, Muna’s chief executive officer, in Muna’s press release (2).

“By applying spatial multi-omics to unique patient phenotypes, Muna's MiND-MAP platform is able to determine the genetic and cellular basis of progression and resilience in neurodegenerative diseases. The alliance exemplifies our discovery ethos, to utilize advanced data and platform tech to identify high-confidence, human-data-derived, causal targets, which we can support with GSK’s scale and expertise in clinical development and commercialization, to bring desperately needed new therapeutic solutions in Alzheimer’s disease,” said Khavandi, in the Muna press release.

1. Relation. Relation Announces Two Strategic Collaborations with GSK to Advance Therapeutics for Fibrotic Diseases and Osteoarthritis. Press Release. Dec. 10, 2024.
2. Muna Therapeutics. Muna Therapeutics Announces Strategic Alliance with GSK to Accelerate Development of Novel Treatments for Alzheimer’s Disease. Press Release. Dec. 5, 2024.

With these agreements, the respective parties will work to advance therapeutic candidates for treating fibrotic diseases, osteoarthritis, and Alzheimer’s disease.