Feliza Mirasol is the science editor for 

Feliza Mirasol

Clinical-stage biotechnology company Alentis Therapeutics (Alentis), which specializes in developing treatments for Claudin-1 positive (CLDN1+) tumors and organ fibrosis, announced that it has raised $181.4 million in Series D financing. The financing was supported by a syndicate of top-tier biotech investors. OrbiMed led the way, with Novo Holdings and Jeito Capital as co-leads. This financing will support Alentis’ efforts to develop a significant pipeline of CLDN1-targeted antibody-drug conjugate (ADC) drugs for treating solid tumors, according to a Nov. 12, 2024 press release (1).

In addition to the lead investors, new investors such as Frazier Life Sciences, Longitude Capital, Catalio Capital, Piper Heartland Healthcare Capital, and Avego Bioscience Capital contributed to the financing, while existing investor RA Capital Management also provided significant backing, along with support from Morningside Venture Investments, BB Pureos, Bpifrance (via its InnoBio 2 fund) as well as other early institutional investors.

“This financing is a testament to the transformational potential of CLDN1 [ADCs] for the treatment of solid tumors,” said Roberto Iacone, chief executive officer of Alentis, in the company press release. “Let me take this opportunity to extend a warm welcome to our new investors. We’re excited to execute our development strategy and deliver clinical data for our programs over the next 12–18 months.”

The company intends to use proceeds from the financing to conduct Phase I/II clinical trials of ALE.P02 and ALE.P03, two ADCs targeting CLDN1, as well as conduct further development of its pipeline. Some of the proceeds will go toward general corporate purposes. FDA cleared an investigational new drug application for a Phase I/II clinical trial of ALE.P02 (with a tubulin inhibitor) to study the ADC candidate in advanced or metastatic CLDN1+ squamous solid tumors. This trial is expected to begin in the first quarter of 2025. Meanwhile, ALE.P03 (with a topoisomerase I inhibitor) is expected to begin a first-in-human trial in patients with CLDN1+ tumors in 2025.

“With this financing, Alentis is now well positioned to advance its CLDN1 antibody-drug conjugates, which have the potential to be first and best-in-class therapies for the treatment of cancer,” said Dina Chaya, partner at OrbiMed, in the press release.

“ADCs have shown their potential to be highly effective cancer treatments,” added Naveed Siddiqi, senior partner, Novo Holdings, Venture Investments, in the release. “Alentis has an exciting pipeline of first-in-class ADCs, and we are proud to have co-led this round. We are looking forward to seeing the data generated from the first clinical trials of ALE.P02 and ALE.P03.”

In addition, three new board members have been appointed as part of the financing and will join the Alentis board of directors. The three new board members are Chaya, who has over 20 years of experience in healthcare investments with a focus on biopharma and medical technology; Anna Chen, principal at Frazier Life Sciences, who has been involved in the investment and building of many of Frazier Life Sciences’ biopharmaceutical portfolio companies over the past seven years; and Brian Liu, managing director at Longitude Capital, who brings more than 10 years of biopharma consulting and investing experience, with a focus on transformative healthcare innovation.

1. Alentis Therapeutics. Alentis Therapeutics Raises $181.4 Million in an Oversubscribed Series D Financing to Advance the Clinical Development of Anti-Claudin-1 ADCs in Solid Tumors. Press Release. Nov. 12, 2024.

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Financing from OrbiMed, Novo Holdings, Jeito Capital, and others will enable Alentis Therapeutics to advance its pipeline of Claudin-1-targeted ADCs for treating solid tumors.

Alentis Therapeutics to Advance Anti-Claudin-1 ADCs to Treat Solid Tumors on $181.4 Million Financing

 (Kebilidi, PTC Therapeutics) on Nov. 14, 2024. Eladocagene exuparvovec-tneqan is an adeno-associated virus vector-based gene therapy for treating adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency (1). According to FDA, Kebilidi is the first FDA-approved gene therapy indicated for this disease.

Administration of the gene therapy is performed via four infusions during one surgical session into a large structure in the brain that is involved in motor control. FDA noted in its press release that Kebilidi should be administered in a medical center that specializes in pediatric stereotactic neurosurgery, which is a technique that uses imaging and special equipment to deliver therapies to specific areas in the brain. Following infusion, the treatment leads to expression of AADC and a subsequent increase in the production of dopamine, which is a critical neurotransmitter in the brain associated with movement, attention, learning and memory, according to the press release.

“Clinical advancements in the field of gene therapy continue to lead to the discovery and availability of innovative treatment options for rare diseases that are otherwise difficult to manage,” said Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research (CBER), in the release (1). “Today’s approval underscores our commitment to help make safe and effective treatments available for patients in need.”

AADC deficiency is a rare genetic disorder that affects the production of some neurotransmitters, interfering with the body’s nervous system cells’ ability to communicate with each other. Symptoms include delays in gross motor function (head control, sitting, standing, and walking), hypotonia (weak muscle tone), and developmental and cognitive delays.

“AADC deficiency can cause a range of debilitating symptoms, including life-threatening complications,” said Nicole Verdun, MD, director of the Office of Therapeutic Products in CBER, in the release (1). “[The] approval represents important progress in the advancement and availability of safe and effective treatments for debilitating genetic disorders.”

Eladocagene exuparvovec-tneq’s safety and effectiveness were demonstrated in an open-label, single-arm clinical study with 13 pediatric patients who had confirmed diagnoses of AADC deficiency. All patients at the start of the study were noted as having no gross motor function (the most severe presentation of AADC deficiency) and decreased AADC activity in the plasma. The patients treated with eladocagene exuparvovec-tneq were compared to untreated patients (natural history). At week 48 following treatment, motor milestone assessments were completed for 12 of the 13 patients. The efficacy of the gene therapy was demonstrated by evidence of improved gross motor function in eight out of 12 patients treated that was not reported in untreated patients with the severe presentation of AADC deficiency.

Eladocagene exuparvovec-tneq underwent an accelerated approval process, and its application received priority review, orphan drug designation, and was granted a rare pediatric disease priority review voucher by FDA. Continued approval for the AADC deficiency indication may be contingent upon the agency receiving verification and description of the clinical benefit of the product, such as the durability of improved symptoms, in a confirmatory clinical trial. Eladocagene exuparvovec-tneq is in an ongoing confirmatory trial to verify its clinical benefit.

In addition to the gene therapy product’s approval, FDA also authorized an infusion tube, the SmartFlow Neuro Cannula (ClearPoint Neuro) (2), which is inserted into a target in the brain (parenchymal tissue), for delivering eladocagene exuparvovec-tneq. FDA stated in its release that the SmartFlow Neuro Cannula is currently the only FDA authorized device indicated for use in the administration of eladocagene exuparvovec-tneq.

FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency

. Press Release. Nov. 14, 2024.
2. ClearPoint Neuro. ClearPoint Neuro Announces FDA De Novo Marketing Authorization of SmartFlow Cannula for Direct Delivery of Gene Therapy to the Brain. Press Release. Nov. 13, 2024.

FDA's approval of eladocagene exuparvovec-tneq (Kebilidi) makes that product the first FDA-approved gene therapy for treating AADC deficiency.

PTC Therapeutics Gains FDA Approval for AADC Deficiency Gene Therapy

Frontier Scientific Solutions, a temperature-controlled storage and transportation company that exclusively serves the life sciences industry, announced on Nov. 13, 2024 that it has established state-of-the-art current good manufacturing practice (CGMP) facilities at Wilmington International Airport (ILM) in North Carolina, United States, and Shannon Airport (SNN) in County Clare, Ireland. The strategic locations will enable the company to create the first life sciences-dedicated direct global gateway, according to a company press release. Supported by a $1.5 billion commitment from an investor, the gateway is central to Frontier Scientific Solutions’ goal to create streamlined shipping lanes—starting with a route between North America and Europe—specifically for time- and temperature-sensitive pharmaceutical and biopharmaceutical products.

These facilities are supported by the $1.5 billion funding from GID, a real estate investor, owner, operator, and developer. Frontier Scientific Solutions expects that the facilities in both locations will bring further financial and supply chain management benefits through designation as Free Trade Zones (FTZ). The company unveiled plans at ILM for the scale and impact of its global gateway model. The company expects that this model will enable companies in the life sciences industry to transport time- and temperature-sensitive materials in significantly shorter timeframes. Benefits include fewer touchpoints, less risk, and more sustainable operations.

"We are thrilled to announce this transformative partnership with the airports at Wilmington and Shannon, enabled by GID's investment,” said Steve Uebele, CEO of Frontier Scientific Solutions, in the company press release. “Our initiative disrupts the life science supply chain market—promoting zero product losses, increasing sustainability, and directly saving lives—and it marks the first step in Frontier's goal to become the market-leading solutions provider."

The funding will be used for the construction of CGMP-compliant, temperature-controlled storage facilities housed at Shannon and within the FTZ at Wilmington. Having FTZ designation will facilitate streamlined customs and FDA processing, duty deferral, and operational efficiencies, all of which are critical for the efficient and secure distribution of life sciences products across international borders, the company stated in the release.

"GID’s commitment underscores our dedication to developing cutting-edge infrastructure that meets the specialized needs of the life sciences industry,” said Greg Bates, president and CEO of GID, in the release. “The new facilities in Wilmington and Shannon will create an unparalleled logistics network between the US and Europe, empowering companies to deliver critical healthcare products with unmatched speed and precision."

Based on live case studies conducted by Frontier Scientific Solutions, it was consistently demonstrated that a reduction of days to weeks in the transportation and waiting times for temperature-sensitive shipments were achieved with its gateway model. “Providing the industry with direct distribution makes this global gateway model faster, reducing exposure to temperature excursions, risk from unnecessary touchpoints, and carbon emissions. It will set a new benchmark for quality, speed, and environmental responsibility,” the company stated in the press release.

The facilities at ILM and SNN will be linked by a direct lane dedicated exclusively to life sciences materials. Flight operations are slated to begin early in 2025. Frontier Scientific Solutions also offers a fast direct-ocean lane from North Carolina to Cork, Ireland, and can achieve delivery in 12 days via this route. This new infrastructure will allow the company to disrupt the market by enabling faster deliveries while reducing reduced risk.

In addition to supply chain logistics, the biopharma industry is seeking ways to minimize risks in providing temperature-sensitive molecules to patients, such as 

innovating product formulation and drug delivery systems

Prioritizing Formulation Strategies for Temperature-Sensitive Biotherapeutics

Pharmaceutical Technology Trends in Formulation

Frontier Scientific Solutions has unveiled plans to use a $1.5 billion commitment from a major investor to create a global free trade zone gateway to facilitate the life sciences supply chain using Wilmington International Airport in North Carolina.

Frontier Scientific Solutions Plans a Global Free Trade Zone Gateway to Supply Life Sciences Industry on Back of $1.5 Billion Investment

Avantor, a provider of mission-critical products and services to the life sciences and advanced technology industries, announced on Nov. 14, 2024 that it has opened its new flagship Bridgewater Innovation Center in Bridgewater, NJ.  Spanning 60,000 square feet, the state-of-the-art R&D facility joins the company’s network of 13 research and innovation centers globally.

The expanded facility doubles the company’s previous laboratory and pilot plant capacity and was purpose-designed to enable collaborative work. The center houses spaces for upstream and downstream process development, dedicated analytical testing labs, and a viral vector laboratory. It also houses an expanded pilot plant that supports scale-up simulations, which is intended to enable rapid customization across the entire bioprocessing workflow, according to a company press release.

“Our new Bridgewater Innovation Center is twice the size of our previous facility and adds capacity, talent, and capabilities to support the growing demands for monoclonal antibodies [mAbs], cell and gene therapy [CGT], and mRNA [messenger RNA] workflows,” said Michael Stubblefield, president and CEO of Avantor, in the press release. “Working side-by-side with customers in our network of innovation centers across the globe enables us to power science at every step to deliver life-changing therapies faster and more cost effectively.”

The center gives Avantor the capabilities to optimize and accelerate biomanufacturing processes at scale. Advantages include faster problem solving, streamlined knowledge exchange, and strategic co-innovation. The facility is staffed with PhD scientists, bioengineers, biologists, and process engineers, which enhances capacity and expertise to support customers' needs. As stated in the press release, “The facility also supports Avantor’s sustainability goals, reinforcing the [c]ompany’s commitment to environmental responsibility.”

The center is aimed at helping biopharmaceutical companies accelerate the development of vital therapies across multiple modalities, including mAbs, CGTs, and protein/peptide therapeutics. Expansive collaboration spaces built throughout the facility allow scientists, biologists, and bioengineers to work side-by-side with biopharma manufacturers. These collaborations leverage the company’s advanced products, real-time analytical data, and process knowledge to optimize processes and materials. Having this co-development environment saves crucial time and resources, according to the company in the press release.

® (1), Benoit Gourdier, executive vice-president and head, Bioscience Production Segment, Avantor, noted how the pace of innovation in biopharma has been posing challenges, particularly in relation to which platform technology to use. “This is something where we’re seeing a fast evolution. From that perspective, at Avantor we are agnostic because we can provide product, solutions, technologies, platforms, support, and this innovation center is a good example. No matter the modality, we have a solution for the players,” Gourdier said in the interview.

Nandu Deorkar, senior vice-president, Bioscience Production Research & Development, Avantor, also spoke in the interview and pointed out that, because of the shift to personalized medicine manufacturing, there will be more distributed manufacturing. “If you look at the cell and gene therapy [space], [patients] will be treated on a personal basis, so there will be more distributed manufacturing on a local basis; so how do we support this geographically?” Deorkar said in the interview (1).

Meanwhile, the anticipated flood of new biotherapeutic modalities onto the market in the near future aimed at precision treatment is expected to open up many new opportunities for service providers, Gourdier said in the interview (1).

Avantor Sees Future of Opportunities with Growing Development of Next-Gen Biotherapeutics

Avantor's newly launched expanded Innovation Center in Bridgewater, NJ, is designed for integrated workflows and features purpose-built collaboration spaces. 

Newly Opened Innovation Center in New Jersey Gives Avantor Advantage in Solving Life Sciences Challenges

In preparation for the launch of its new innovation center in Bridgewater, NJ, Avantor executives discussed a future full of opportunities for service providers, which will come from a growing number of next-generation biotherapeutics in the development pipeline that are anticipated to reach the market in the near future.

“The first thing [that comes to mind] is lots of opportunities, because this is really going back to the base of innovation,” said Benoit Gourdier, executive vice-president and head, Bioscience Production Segment, Avantor, in an interview with 

® at a press event held at the Bridgewater facility on Nov. 13. 2024.

Where once the biopharma industry was dominated by monoclonal antibodies (mAbs), the industry can now expect to see a wave of newer, more innovative therapies aimed at achieving precision treatment. “Starting 25-30 years ago, it was really mAbs, mAbs, mAbs, and traditional vaccines,” Gourdier said, adding, “We grew up in this environment. Now we have this diverse portfolio of modalities, so [that will provide] lots of opportunities to go after, to learn.”

The challenges that Gourdier anticipates in the future could likely revolve around chemistry, fluid handling, meeting high purity in a regulated market, among others, but Gourdier is confident that Avantor will be well prepared to meet these challenges and to offer the appropriate support as a service provider.

Nandu Deorkar, senior vice-president, Bioscience Production Research & Development, Avantor, added that, because of the shift to personalized medicine manufacturing, there will be more distributed manufacturing. “If you look at the cell and gene therapy [space], [patients] will be treated on a personal basis, so there will be more distributed manufacturing on a local basis; so how do we support this geographically?” Deorkar said in the interview.

Deorkar also added, “Some of these therapies have 48 hours to 72 hours injection requirement after manufacturing, so [not all] the manufacturing can be done [in one place].”

Gourdier, meanwhile, pointed out that, in addition to the expectation of a different manufacturing and supply chain scenario for next-gen biotherapeutics, the industry suffered from supply chain disruptions due to the COVID-19 pandemic, which are still ongoing in the post-COVID environment. Regionalization has become more important, he noted. “[Developers] want global partners with regional focus,” he stated.

Other factors that have disrupted the pace of development for these next-gen biotherapeutics has been a drop in funding as a direct result of the COVID-19 pandemic, Gourdier added. “Most of the big players are okay,” he observed, “but for smaller players, the amount of money available for them has decreased significantly. We are just [coming] back [from that]. Now we are in modest recovery from that (i.e., the funding) perspective.”

Meanwhile, the pace of innovation has itself been posing challenges, particularly in relation to which platform technology to use. “This is something where we’re seeing a fast evolution. From that perspective, at Avantor we are agnostic because we can provide product, solutions, technologies, platforms, support, and this innovation center is a good example. No matter the modality, we have a solution for the players,” Gourdier stated.

Avantor’s new Bridgewater Innovation Center is set to launch on Nov. 14. It has been designed as a state-of-the-art research and development facility and joins the company’s network of 13 research and innovation centers globally.

Executives at Avantor talk about the future of the biopharmaceutical industry and the anticipated impact that a wave of next-generation biotherapeutics will bring.

Ginkgo Bioworks, a provider of a cell programming platform and biosecurity, announced on Nov. 12, 2024 that it has completed the first milestone of a previously announced partnership (1) with Merck, known as MSD outside of the United States and Canada. Their collaboration aims to improve biologics manufacturing, and, according to the terms of their agreement formed in August 2023, Ginkgo will receive a research milestone payment of $9 million in cash for completing this milestone.

Under their partnership, Ginkgo uses its expertise and capabilities in cell engineering, ultra-high-throughput multiplexed screening, protein characterization, and process optimization to improve biologics production efficiency and increase yields. The company is eligible to receive up to $490 million in aggregate of upfront research fees, research milestone fees, option license payments, and commercial milestone payments (1).

"We're excited to achieve the first milestone of our collaboration with Merck and continue our work to improve biologic manufacturing production," said Jason Kelly, CEO and co-founder, Ginkgo Bioworks, in a company press release. "It's very meaningful when our team can drive impactful and technically innovative work like this, especially when we're working shoulder-to-shoulder with counterparts like the team at Merck.”

The two companies will now move on to the second stage of the partnership, which involves focusing on the continued improvement of the biologics production process.

“As we go into the next phase of this project, we'll continue to deploy the strain engineering, high-throughput screening, and process development capabilities as well as the assets and know-how that make Ginkgo a unique and powerful partner to top pharmaceutical companies," Kelly said in the release.

The August 2023 partnership deal is the second collaboration between Ginkgo and Merck. In October 2022, the companies entered into a biocatalysis project to improve Merck's API manufacturing (2). Under the biocatalysis deal, Ginkgo earned an upfront research and development fee and was eligible for success-based research and development milestone payments. Ginkgo was also eligible to earn commercial milestone payments for each of a specified number of biocatalysis targets. These targets have the potential to total, in the aggregate, up to $144 million, according to a company press release issued at that time.

"At Merck we are always seeking new and innovative ways to optimize process efficiency while maintaining product integrity," said Michael Kress, PhD, senior vice-president, development sciences and clinical supply, Merck Research Laboratories in an Aug. 7, 2023 press release (1). "We are pleased to build upon our relationship with the Ginkgo Bioworks team."

1. Ginkgo Bioworks. Ginkgo Bioworks Announces Collaboration with Merck to Improve Biologic Manufacturing. Press Release. Aug. 7, 2023.
2. Ginkgo Bioworks. Ginkgo Bioworks Announces Collaboration with Merck to Improve Active Pharmaceutical Ingredient Manufacturing. Press Release. Oct. 11, 2022.

Under an ongoing partnership with Merck, known as MSD outside of the United States and Canada, Ginkgo Bioworks has achieved the first milestone in a project aimed at improving biologics production.