The mRNA vaccine facility will rely on BioNTech’s “BioNTainers,” modular units that can be set up quickly and adjusted to produce different mRNA vaccines. Built to be flexible and scalable, it meets health challenges while helping expand the local vaccine network. If successful, the Kigali site could become the continent’s first commercial mRNA vaccine manufacturing facility.

“This manufacturing site is about empowering Africa with the tools and expertise to tackle health challenges independently,” said Karl Nehammer, EIB Vice-President responsible for health. “By working with BioNTech and the European Commission, we’re supporting a future where vaccines are produced in Africa, for Africa. This partnership is a major step forward for health, jobs, and innovation across the continent.”

The facility is expected to not only produce vaccines for widespread use but also manufacture clinical trial materials for local partners, supporting skills development and job creation while strengthening Rwanda’s position as a hub for medical innovation. “We recognize that the challenges in global health are too vast for any single entity to solve alone. BioNTech is dedicated to working across the entire development chain, partnering with local communities, researchers, governments, and not-for-profit organizations to make a meaningful impact. The support by the European Commission, European Investment Bank and CEPI are an important contribution to the joint efforts of advancing and strengthening the implementation of a local mRNA vaccine ecosystem – covering the spectrum from clinical trials to commercial production,” said Sierk Poetting, Chief Operating Officer of BioNTech.

The initiative reflects close collaboration between BioNTech, the Coalition for Epidemic Preparedness Innovations (CEPI), and Team Europe partners, EC and EIB. It aligns with the EU’s Global Gateway strategy and supports the African Union’s goal of producing 60% of the continent’s vaccines domestically by 2040. Jozef Síkela, Commissioner for International Partnerships, said: “Global health is a key priority of the Global Gateway strategy, and the EU has already invested more than €1.9 billion in local vaccine and medicine manufacturing and affordable access in Africa. The agreement with BioNTech to support the advancement of its state-of-the-art mRNA manufacturing facility in Rwanda will boost expertise across the region and build increased independence from entities outside of Africa.”

CEPI’s backing, including a grant of up to €130 million announced in 2024, is intended to ensure that vaccines produced at the Kigali site remain accessible and affordable. Dr Amadou Sall, Executive Director of Manufacturing and Supply Chain at CEPI, added: “Establishing the pioneering Rwanda facility as the first mRNA vaccine manufacturing site in the region following the globally recognised Goods Manufacturing Practice will boost regional vaccine capacity to help more rapidly respond to public health threats, including new outbreaks that strike locally. The project will make an important contribution to Africa CDC’s goal for 60% of the vaccines needed by the continent to be produced on the continent by 2040, strengthening regional health security and global pandemic preparedness.”

With a compound annual growth rate (CAGR) of 22.4%, the present bioprocessing market is projected to be worth more than $218 million in 2023 and is expected to reach more than $599 million by 2028, according to one of the market research studies published. The main causes of this development are the rising demand for biopharmaceuticals, the increasing acceptance of continuous bioprocessing among contract manufacturing companies (CMOs) and contract manufacturing organisations (CMOs), and the benefits of continuous bioprocessing over batch and fed-batch modes of production.

Knowledge of Market Dynamics By use of PAT’s application to optimise continuous bioprocesses

Process analytical technology (PAT) is the name given to a system including analytical tools meant for monitoring and control of industrial processes. Apart from sensor technologies, tools for process analysis and testing (PAT) such as spectroscopy and chromatography enable continuous monitoring of important process parameters (CPPs) and critical quality attributes (CQAs) in real time. This helps manufacturers to quickly spot any kind of deviation and implement the required corrections to maintain the integrity of the process as well as the quality of the goods. The PAT advances and helps the producers maximise their operations in several spheres, including the following:

Process knowledge and control: PAT helps to deepen understanding of the bioprocessing environment by means of insights on links between the process parameters and product characteristics. This helps one to grasp the bioprocessing environment better. Manufacturers may make use of this knowledge to create advanced control systems including feedback control loops or even model predictive control, therefore optimising the process performance and ensuring consistency of the product.

By means of PAT-enabled optimisation, which simplifies bioprocessing operations, it is feasible to simplify opportunities for process intensification as well as efficiency gains, thereby reducing cycle durations and increasing productivity. By always monitoring and modifying process parameters, manufacturers may minimise cycle times, boost throughput, and even raise general productivity. This enables their progressive progress and goal attainment.

PAT is in line with the ideas that apply to quality by design (QbD) since it helps to build solid processes that go on to produce things regularly with the necessary quality traits. This enables manufacturers to go forward and include quality into the process from the very start, therefore lowering the risk related to product failures and deviations. One achieves this by including PAT instruments into the phases of design and development.

Apart from a cut in waste, production expenses also reduce. By means of consistent monitoring and control made available by PAT, one may help identify and minimise process inefficiencies, therefore lowering the raw material, energy, and waste generation amounts. Consequently, not only does this save production costs, but it also aids in the accomplishment of sustainability targets by lessening environmental negative effects.

Regarding the facilitation of regulatory compliance, PAT gives manufacturers the tools and the data they need to present to regulatory authorities demonstrating that they have a complete awareness of the process, that they have control over it, and that they are consistent with it. Using PAT helps producers to simplify regulatory filings, speed up product approvals, and guarantee compliance with strict regulatory criteria.

All things considered; PAT-enabled optimization has a significant potential in the continuous bioprocessing industry. This possibility motivates manufacturers to achieve higher degrees of process efficiency, quality, and regulatory compliance, therefore generating more revenues.

In terms of ongoing bioprocessing, high production and cost promote chromatography system appeal.

Product into chromatography systems with filtering systems along with devices; consumables; bioreactors; cell lines, cell culture medium, buffers, and reagents; also, other products divide the market for consistent bioprocessing. This is something one ought to give thought. Among other components, the chromatography systems and the consumables comprise resins, membranes, buffers, solvents, columns, reagents, and other consumables like autosamplers, fittings, and tubing detectors. Consistent chromatography techniques are absolutely essential for continual downstream bioprocessing if one is to get high protein purity. These advanced techniques offer a lot of interesting possibilities. One can keep the process running constantly by running several chromatography columns either countercurrent or even concurrent. This is so because the loading is done in the first column and all the other subsequent processes—elution, regeneration, washing, and re-equilibration—occur inside the next columns. Many chromatographic methods are part of the continuous mode of operation. Countercurrent chromatography (CCT), multicolumn countercurrent solvent gradient purification chromatography (MCSGP), simulated moving bed (SMB) chromatography, and continuous annular chromatography (CAC) among these methods.

Growing demand for biologics has led to the necessity for intensification of the upstream bioprocess in order to raise production and minimise manufacturing costs. This is so due to the growing biologics market. Several studies by the National Centre for Biotechnology Information (NCBI) show how well integrated continuous bioprocessing is applied in manufacturing monoclonal antibodies (mAbs). These studies have indicated that the technique works. For example, one-column continuous chromatography (OCC) and perfusion bioreactor culture using alternative tangential flow technology (ATF) allowed a researcher to get about an eighty percent boost in productivity.

Moreover, companies are fast shifting their focus to chromatography systems in order to fulfil growing industry needs and speed their manufacturing processes. Following that, Waters Corporation and Sartorius AG announced in June of 2023 their cooperation to create integrated analytical solutions for the biomanufacturing process occurring deeper downstream.

The value of approaching farther downstream

Aimed at isolating and purifying the expected biopharmaceutical product from the complex mixture related to cellular components, media, and contaminants generated during upstream production, downstream processes have a sequence of purification steps along with separation techniques. Along with accessories and other products connected with them, these activities sometimes produce items such cell filtration systems, devices, chromatography systems, and consumables. Particularly continuous chromatography systems are reinventing downstream purification by enabling continuous separation as well as the purification of biopharmaceutical products with exceptional precision and throughput. This represents a major advancement in the downstream goods purification process. Large biopharmaceutical companies have effectively applied both aqueous two-phase extraction, also known as ATPS, and periodic countercurrent chromatography, also known as PCC, continuous CTC, MCSGP, and SMB for the aim of continuous capture from the process development scale all the way up to the manufacturing scale. These businesses have been able to avoid the possible process bottlenecks this suggests. These experts help to enhance the cost of the goods, the quality of the output, and the effectiveness of the process. Two among the several resins used in the Protein A resin screening process are MabSelect Sure PCC-Cytiva and Poros ProA-Thermo Fisher Scientific. These resins are reachable all during the process’s continuous capture phase. Apart from compound development and manufacturing companies (CDM), it is expected that more mid-sized biotech companies will arise in the next few years.

Some of the elements driving the high share of the segment in the market are the growing demand for biopharmaceuticals; rising technological advancements like single-pass tangential flow filtration and multicolumn chromatography; the increasing need for intensification of the downstream bioprocess as a result of an increased titer; and lowering production costs in the case of biosimilars and innovator drugs. These elements help the segment to have a significant market share.

An Application-Based Study of the Market for Ongoing Bioprocessing

Among the applications included in the consistent bioprocessing market segmentation are mAbs, vaccines, cell and gene therapy, and other ones. Application drives this segmentation of the market. It so happens that one of the most important subgroups of biotechnology medicine is monoclonal antibodies. The growing pharmaceutical research and development drug pipeline, the increasing emphasis on continuous bioprocessing in the manufacturing of monoclonal antibodies, the expanding clinical pipeline of monoclonal antibodies, and the growing regulatory approvals pertaining to therapeutic antibodies help to explain both the great share as well as the high growth rate inside this segment. These elements taken together have helped the segment to flourish.

Continuous bioprocessing is rapidly gaining speed in monoclonal antibody bioprocessing, so it has the potential to offer several advantages like smaller facility footprints, less investment costs, more flexibility, and economies of process. Once mammalian cell-derived monoclonal antibodies (mAbs) became commercially successful, demand for breakthrough single-use bioreactor systems surged. These technologies can reduce prices and provide far better degrees of flexibility and productivity. The successful proving of the viability of a completely integrated continuous process from the pilot size bioreactor to the therapeutic substance has led to research that has opened the road for its larger application within the industry.

Two more elements driving the growth of the market within this specific category are the increasing frequency of cancer and the growing necessity for cancer therapies. Monoclonal antibodies (mAbs) on the other hand have less side effects than chemotherapy. Another important factor driving the growth of the market is the emergence of new, more efficient and effective monoclonal antibody (mAbs) classes, such anti-PCSK9 monotherapy. Among the most effective monoclonal antibodies (mAbs), including Humira, Rituxan, Avastin, and Pembrolizumab-Keytruda, some have patents set to expire in the next few years. Patents have been lost, hence biopharmaceutical companies have been forced to proceed with including monoclonal antibodies (mAbs) into their drug manufacturing process. Regarding affordable solutions like continuous bioprocessing, demand is predicted to rise given the expanding pharmaceutical drug pipeline as well as the increasing number of regulatory approvals for the modular drug delivery systems (mABs).

Final Thoughts

Now leading the front stage in innovation is the constant bioprocessing sector, which will help to revolutionise manufacturing processes for pharmaceuticals worldwide. This is something one should give careful thought. Consistent bioprocessing provides advantages unmatched in terms of efficiency, productivity, and quality control when compared to conventional batch methods. This is so because continuous, smooth production flow of consistent bioprocessing is what drives An increasing demand for biopharmaceuticals, the rise of integrated end-to–end continuous bioprocessing, government and regulatory initiatives for favourable innovative technologies, and a rising acceptance among both contract manufacturing organisations (CMOs) and contract manufacturing organisations (CMOs) are driving the market under several angles. Indeed, the acceptance of continuous production techniques by pharmaceutical manufacturers is causing the market to develop fast and show diversity. Beginning with the upstream cell culture and fermentation operations and working all the way down to the downstream production processes of purification and formulation, continuous bioprocessing systems are applied across the whole biopharmaceutical manufacturing process. Furthermore adding to the improvement of process control and optimization—which finally yields higher efficiency and guarantees regulatory compliance—process analytical technology, sometimes referred to as PAT, in addition to automation.

These data demonstrate immune-stimulatory activity in solid tumor patients with highly-refractory malignancies, including patients who have failed prior checkpoint inhibitors. Also presented were pre-clinical data establishing the immune-modulatory and anti-tumor effects of RGX-104. The company presented the data at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Philadelphia.

RGX-104 is a liver X receptor (LXR) agonist that upregulates the expression of the target gene, Apolipoprotein E (ApoE), triggering several downstream effects via ApoE receptors. In pre-clinical data presented today, treatment with RGX-104 in mouse models resulted in dual effects on myeloid-derived suppressor cells (MDSCs) and dendritic cells (DCs), both innate immune cells that play a central role in regulating anti-tumor immunity and response to checkpoint inhibitors. Innate immune activation with RGX-104, coupled with a reduction in tumor blood vessels, resulted in anti-tumor activity as a monotherapy as well as synergy with checkpoint inhibitors (CPI) in several drug-resistant mouse models. These data provide rationale for Rgenix’s ongoing Phase 1a/b trial of RGX-104 in advanced cancer patients and support evaluation of RGX-104 as both a monotherapy as well as in combination with CPIs.

As part of the ongoing Phase 1a/b clinical trial, 15 patients with a variety of solid tumors have been treated with escalating doses of RGX-104 monotherapy. Patients treated with RGX-104 had a median of six prior therapies with a range of 1-12, highlighting a population of patients with profoundly resistant disease.

Activation of the LXR-ApoE pathway with oral administration of RGX-104 was associated with immune-stimulatory activity in 9 of 10 evaluable patients. This was demonstrated by an increase (up to 11-fold) in activated circulating PD-1+CD8+ T cells during treatment. T cell activation was observed in patients who experienced modulation of the innate immune system during treatment. The effect of RGX-104 on the innate immune system consisted of both MDSC depletion (up to 95% decrease) as well as DC activation as indicated by induction of PD-L1 expression (up to 100% increase). In most cases these effects were observed within two weeks of treatment initiation and generally preceded the onset of T cell activation.

Safety data demonstrate good tolerability with on-target safety findings in the first three dosing cohorts. One patient experienced a DLT of grade 4 reversible neutropenia – a known potential effect of LXR agonism – that reversed within one week, allowing the patient to subsequently tolerate a 50% dose reduction. No MTD has been reached to date. Stable disease has been observed in 4 of 12 evaluable patients, including three who have failed prior checkpoint inhibitor therapy, for periods of at least 8 weeks.

“We are very pleased to see robust evidence of immune stimulation in such highly-pretreated patients,” said Roger Waltzman, MD, MBA, and Chief Medical Officer of Rgenix. “CPI therapy is now commonplace but only a minority of patients derive clinical benefit. We hope the effects of RGX-104 on modulating barriers to innate and adaptive immune function will enable a larger number of patients to benefit from this therapy. These preliminary results also highlight the potential for development of RGX-104 as a monotherapy.”

Rgenix plans to enroll subsequent dose-escalation cohorts of the RGX-104 monotherapy trial in Q4 2017. Additionally, Rgenix is planning to initiate the Phase 1b expansion component of the study, comprised of disease directed cohorts receiving RGX-104 monotherapy as well as cohorts receiving RGX-104 combined with a CPI, projected to begin in 1H 2018.

“These preliminary data establish RGX-104 as a potential first-in-class oral immunotherapy agent with broad immune-stimulatory activity and a unique dual mechanism targeting innate immunity,” said Masoud Tavazoie, MD, PhD, and Chief Executive Officer of Rgenix. “These results also further validate our discovery platform at Rgenix, as well as our pipeline of other drug candidates slated to begin entering clinical-stage development in 2018.”

The LXR-ApoE pathway was discovered as a cancer target using a microRNA (miRNA) based target discovery approach originally developed at The Rockefeller University and now exclusively licensed to Rgenix.

About Rgenix

Rgenix, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancer progression. The company is pursuing several first-in-class drug candidates to treat cancers of high unmet need. Rgenix identifies novel cancer targets using a microRNA based target discovery platform originally developed by Rgenix’s scientific co-founders at The Rockefeller University and now exclusively licensed to Rgenix. The company brings together distinguished scientific founders, a seasoned Board, and a leadership team comprised of experienced drug developers. The company is funded by leading biotechnology investors, including Novo A/S, Sofinnova Partners, and Alexandria Venture Investments. For more information, please visit www.rgenix.com

Contacts
Media:
RooneyPartners
Marion Janic, 212-223-4017
mjanic@rooneyco.com

SPCI is developing Altemia™ as a treatment for pediatric patients with sickle cell disease.

The Company previously announced enrollment completion of the clinical trial, highlighting high retention rates and the possibility for patients to enroll into an open label extension that will collect long term safety data while continuing to monitor the effectiveness of the drug.

“We are excited that database lock, another key milestone for the study, is being achieved. We will discuss progress made in our sickle cell research initiatives with the scientific community at SCDAA. We expect to present all data available at future scientific events. Altemia™ has the potential to be an important new treatment option for people with sickle cell disease who experience devastating vaso-occlussive events, which can be extremely disruptive in their lives”, said Dr. Frederick D. Sancilio, President and Chief Executive Officer of SPCI.

About Sickle Cell Disease (SCD)
Sickle Cell Disease (SCD) is a group of genetic disorders that results in dysfunctional hemoglobin (HbS) and a depletion of certain lipids in the walls of blood cells. These abnormalities create an inflammatory state and an increase in the red and white blood cell’s tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop organ damage and strokes. There are approximately 100,000 cases of SCD in the United States. Treatment options are limited. The cost of care for this group may exceed $5BB.

About Sancilio Pharmaceuticals Company, Inc.
Sancilio Pharmaceuticals Company, Inc. (SPCI) is a fully integrated, specialty pharmaceutical company focused on developing, manufacturing and commercializing pharmaceutical products, including those based on our proprietary Advanced Lipid Technologies® (ALT®) platform. SPCI is pursuing treatments for sickle cell disease, short bowel syndrome and severe hypertriglyceridemia. We utilize our cGMP compliant facility to develop and manufacture our products. Our ALT® platform is designed to enhance the bioavailability, reduce the food effect and improve the efficacy of lipids and lipophilic active pharmaceutical ingredients (APIs). Lipids are hydrophobic or amphipathic molecules, including fatty acids, steroids (including hormones) and fat-soluble vitamins (such as vitamins A, D, E and K). Our business model is to apply our ALT® platform to lipids or lipophilic APIs to create unique product candidates that address the disorders and diseases resulting from imbalances of lipids in the body. In addition to our primary focus of developing our proprietary products using the ALT® platform, we make use of, and license rights to, our proprietary ALT® platform and other technologies to third parties, providing both development and subsequent soft gelatin encapsulation services. More information is available at: www.sancilio.com

Contacts

Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer

Antibodies to PNAG have the potential to prevent many serious and life-threatening infections such as pneumonia, meningitis, bloodstream infections, gonorrhea, and also those caused by antibiotic resistant bacteria such as MRSA. The World Health Organization has warned that the increasing development of numerous antibiotic-resistant “superbugs” pose an enormous threat to human health. Based on these results, Alopexx plans to initiate a Phase 2 clinical study with AV0328 in the first quarter of 2018.

“We are very encouraged by these early clinical data demonstrating the safety, tolerability and initial indications of clinical activity of AV0328 in humans,” said Hal Landy, M.D., Chief Medical Officer at Alopexx. “We look forward to further evaluating AV0328 against a breadth of infections caused by PNAG-expressing pathogens, including serious soft-tissue infections caused by methicillin-resistant S. aureus (MRSA), pneumonia, meningitis, tuberculosis and sexually transmitted diseases, including those caused by antibiotic-resistant organisms.”

“Our modified, synthetic vaccine that targets natural PNAG expressed on the surface of most pathogenic bacteria, as well as important eukaryotic pathogens like malaria and numerous fungi, could represent a new paradigm for disease prevention by inducing protection against a large number of infectious agents, including those manifesting high levels of antibiotic resistance” Gerald B. Pier, Professor of Medicine, Harvard Medical School, Microbiologist, Brigham and Women’s Hospital.

About the Phase 1 Clinical Study
The Phase 1 clinical study was designed to evaluate the safety, tolerability, pharmacodynamics (PD) and preliminary efficacy of AV0328 in 16 healthy volunteers across four dosing cohorts ranging from 15 to 150 mg. PD was assessed by measuring the increase and time course of serum titers against PNAG and complement binding (C1q deposition) activity. Clinical efficacy was assessed by measuring killing of PNAG-expressing pathogens by subject sera in vitro.

AV0328 was safe and well-tolerated in this study with only minor and transient injection site reactions observed in each dose group. At the two highest doses (75 mg and 150 mg), clear increases were noted in antibody titers against PNAG, as well as a positive indication of protective immunity, as measured by complement activation and binding to the PNAG antigen. In addition, robust bactericidal killing of N. gonorrhea, including antibiotic-resistant strains, and N. meningitidis (serogroups A, B, C, W, Y), as well as opsonic killing of S. pneumoniae, multi-drug resistant Klebsiella pneumonia, colistin and multi-drug resistant strains of E. coli and S. aureus, including MRSA clinical isolates, was observed.

About AV0328
AV0328 is a synthetic pentameric oligosaccharide of b-1-6-linked D-glucosamine sugars conjugated to tetanus toxoid that elicits a protective antibody response against a large number of microbial pathogens associated with infections. PNAG, which is expressed on the surface of various bacteria, fungi and protozoan organisms, has been shown to be a critical factor in the virulence of many pathogens. In its natural state, PNAG has a profound ability to avoid eliciting an effective immune response. Bacterial strains that lose the ability to produce PNAG generally have a significantly reduced ability to cause infections. The fact that so many pathogens express PNAG suggests that it is a critical evolutionary feature for both microbial survival and for evading the mammalian immune system. A vaccine that effectively overcomes immune evasion to elicit antibodies that kill PNAG-producing pathogens could provide protection against not just individual strains or even an entire species of a pathogen, but against a wide array of serious microbial infectious agents.

About Alopexx Vaccine LLC
Alopexx Vaccine LLC, part of the Alopexx Enterprises portfolio of companies, specializes in the development of vaccines against a range of pathogens and infections. For more information please visit www.Alopexx.com

Contacts:
Christine de los Reyes
(Business Development)
cdelosreyes@alopexx.com
917-319-4915

or

Gina Nugent, Nugent Communications
(Investors and Media)
gina@nugentcommunications.com
617-460-3579

SHAPE (Selective 2′ Hydroxyl Acylation analyzed by Primer Extension), the most widely used approach for probing RNA secondary structure, was developed by Kevin Weeks, Ph.D., and colleagues at the University of North Carolina in 2005.1 SHAPE studies provide scientists with experimental assessment of predicted loops, bulges and pockets in the two-dimensional structures of RNA. The resulting integrated structural prediction enables biological insight into RNA function and detection of ligand binding sites within the RNA.

SHAPEware offers a standardized and modular set of tools for scientists to analyze data generated by SHAPE methodologies and was initially developed by Arrakis to streamline and standardize its own analysis of the SHAPE data that the company is generating in its proprietary drug discovery efforts to design RNA-targeted small molecules (rSMs).

“We are thrilled to share SHAPEware with the broader community of RNA experts,” said Russ Petter, PhD, Founder and Chief Scientific Officer at Arrakis. “The field of RNA structure is advancing rapidly and open-source software allows us to accelerate the best thinking from hundreds of potential users.”

SHAPEware will help researchers identify and evaluate the distinct characteristics of folded RNA through the following features and upgrades:

SHAPEware is designed to be highly modular and customizable, allowing users to evaluate and compare results from different analytic algorithms and SHAPE methodologies.
The initial launch of SHAPEware includes a module to analyze data from SHAPE-MaP (mutational profiling) experiments.

Arrakis continues to develop and adapt novel SHAPE methodologies to study RNA structure. It is anticipated that additional updates and modules will be added.

The SHAPEware open-source software can be downloaded at www.shapewareRNA.com, and the free license requires users to share feedback and any modifications or improvements with the community.

About SHAPE
SHAPE is an acronym for selective 2′-hydroxyl acylation analyzed by primer extension, developed by Kevin Weeks, Ph.D., and colleagues at the University of North Carolina in 2005, and describes the chemical reaction that is used to assess the flexibility of RNA at each nucleotide in any given sequence. This information can be used to establish the secondary structure of an RNA, to monitor structural differences between related RNAs or a single RNA in different states, and to infer ligand binding sites. Several SHAPE-based methodologies have been developed in academic laboratories: SHAPE-Seq, SHAPE-MaP (SHAPE followed by mutational profiling) and icSHAPE (in vivo click selective SHAPE), each requiring different experimental protocols and analysis software.

About RNA-targeted Small Molecules (rSMs)
Emerging insights and technologies focused on understanding the structure of RNA enable the design of RNA-targeted small molecules, or rSMs, a new class of medicines that directly bind and modify the biological function of RNA to treat disease. Unlike conventional drug discovery approaches that focus on proteins, an rSM is designed to modulate function of an individual RNA, and, in the case of mRNA, the expressed protein, by selectively binding to specific, predictable structures in functional regions in the RNA. This new drug discovery approach enables the design of small-molecule drugs that can unlock the therapeutic potential of well-known targets that are not accessible with today’s drugs, as well as identifying new drug targets to intervene in diseases in new ways.

About Arrakis Therapeutics
Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. The company has developed a proprietary platform to identify new RNA targets and drug candidates to treat diseases not addressed by today’s medicines. Arrakis is building a proprietary pipeline of RNA-targeted small molecules (rSMs) focused on neurologic diseases, cancer, and rare genetic diseases.

Arrakis was founded in 2015 by Russell C. Petter, Ph.D., Alan Walts, Ph.D., Henri Termeer and Raj Parekh, Ph.D. with a vision to create a proprietary, transformational discovery platform that identifies small-molecule drugs that act directly on disease-causing RNA. The company was established with seed funding provided by Advent Life Sciences and Henri Termeer. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Mass. Please visit www.arrakistx.com

Contacts

For Arrakis Therapeutics
Kathryn Morris, 914-204-6412
kathryn@theyatesnetwork.com

Avara Pharmaceutical Services has signed an agreement with GSK to acquire a GSK consumer healthcare manufacturing facility in Aiken, South Carolina. “This acquisition is an important component of our strategic plan and expands our services by adding additional solid dose capability in the United States, which is in very high demand,” stated Timothy C. Tyson, Chairman and CEO. The transaction is expected to complete on or before June 1, 2018.

Avara Pharmaceutical Services is a state-of-the-art contract manufacturing and technical services organization providing both API formulation and manufacturing, along with secondary formulation, manufacturing and packaging of small molecule drugs, including highly potent compounds. Avara has secondary manufacturing technologies including granulation, coating, blending, encapsulation, compression and drying of tablets and capsules. Avara also has sterile manufacturing capability.

Following completion of the transaction with GSK, Avara Pharmaceutical Services will have seven sites. Three in the US, including the corporate HQs; one in Puerto Rico, one in the UK, one in Ireland, and one in Italy.

Norwalk, CT (USA) – Corporate HQ
Arecibo, Puerto Rico – Secondary manufacturing and packaging
Shannon, Ireland – API formulation and manufacturing
Norman, OK (USA) – Secondary manufacturing and packaging
Avlon, United Kingdom – API formulation and manufacturing
Liscate, Italy – Sterile Manufacturing.
Aiken, South Carolina – Secondary manufacturing and packaging

“As we celebrate another important milestone, we continue with great confidence to add pharmaceutical services and capabilities with complementary offerings in key regions in this rapidly growing market. Each of our sites have significant professional experience, state of the art capability and a long history of delivering high quality pharmaceuticals that meet or exceed customer expectations and regulatory requirements in every major market around the world. The people who are a part of the Avara team are the key to our long-term success,” said Tyson. “We are focused on delivering on our commitments and earning the trust of every customer we deal with.”

About Avara
Avara Pharmaceutical Services, Inc., based in Norwalk, Connecticut is an international pharmaceutical services company that delivers world-class contract manufacturing and technical services to the pharmaceutical industry. Avara has primary and secondary manufacturing facilities in North America and Europe and supplies products to all major markets around the world. Avara’s broad experience with supply chain, commercialization, product launch and product transfer allow us to sustain exemplary levels of product quality and regulatory compliance. The company is known to exceed customer service level expectations and consistently deliver on time, in full at a fair price.
For more information, please visit our website at www.avara.com

Hoffman La Roche AG completed the construction of phase one of their new vitamins and minerals premix plant at Isando, in the Republic of South Africa, in June 2002. The facility was inaugurated by Dr Marcus Altwegg, head of Hoffman La Roche Vitamins and Fine Chemicals division, on 25 June 2002.

BUSINESS SOLD TO DSM

The business was sold to the Dutch chemicals firm DSM in late 2002, in a deal worth $2.24bn. Early in July 2002 Roche instigated negotiations with DSM for the sale of its entire vitamin and premix manufacturing business. Roche, along with other European companies, was fined in the US and Europe after being found guilty of operating a price-fixing cartel for vitamin and nutritional products.

“Roche was fined in the US and Europe after being found guilty of operating a price-fixing cartel for vitamin and nutritional products.”

The sale went ahead with closure of the deal in October 2003. The sale price negotiated was $2.24bn (€1.750m) and 2.24 million shares in DSM to be issued to Roche. Roche had dropped the price by €200m because of the legal issues surrounding price controlling and also retained liability for court costs and compensation arising from these legal issues.
Roche holds 42% market share worldwide in this business and is the world’s leading supplier of vitamins and carotenoids. DSM had no other vitamin and premix business interest and so avoided monopoly investigations by European government bodies.

ISANDO FACILITY

The facility manufactures vitamin premixes for the food and pharmaceutical industry in South Africa and surrounding African nations, such as Nigeria, Ghana and Kenya. The facility works in cooperation with UNICEF in producing the vitamin premixes necessary to fortify basic foodstuffs in under-developed African nations.

ISANDO PREMIX PLANT PROJECT

Construction of the premix plant at Isando was started in the first half of 2000. The new facility entered full commercial scale production a little later than first planned in 2002. The plant was constructed at an estimated cost of 30m Rand ($2.7m).

This facility was the first phase of a larger project. The subsequent phase, which was completed in early 2004, manufactures vitamin premixes for animal nutrition. The total value of the entire project is estimated at $6m. The capacity of the first phase facility was 1,400t/y. The much larger second phase took production to 3,000t/y by mid-2004.

THE PREMIX MARKET BURGEONS

In the pharmaceutical industry, vitamins are used in supplement preparations such as tablets or capsules. Since companies have begun moving away from using single vitamins to fortify their products and towards multiplicity, the services of premix plants have been in great demand. This has strengthened the premix industry considerably. It is for this reason that a number of premix plant projects have been initiated by leading pharmaceuticals suppliers. These include new Roche premix plants in Poland and at El Salto, Mexico. The company has a total of 48 premix facilities around the world.

Problems have arisen in the vitamins market in recent years due to increased competition from vitamin manufacturers in China who can produce cheaper products and also by the increasing cost of raw materials. The price increases are inevitably linked to the price of crude oil.

TEAVIGO: A RECENT SUCCESS IN SOUTH AFRICA

“DSM is looking to boost revenue by producing products from plant raw materials. A recent success story is Teavigo.”
DSM is looking to boost revenue by producing products from plant raw materials. A recent success story is Teavigo, which is a purified, concentrated form of the green tea active epigallocatechin gallate (EGCG) known as a potent antioxidant. This was developed by Roche prior to the sale to DSM.

Teavigo has achieved GRAS (generally regarded as safe) status from the US FDA and is now being incorporated into a wide range of foods. Prior to this the compound was incorporated into commercial food stuffs in South Africa.

COMBATING THE HIDDEN HUNGER PHENOMENON

Vitamin and mineral deficiencies prevent some 30% of the world’s population from achieving their full mental and physical potential. This phenomenon is often referred to as ‘hidden hunger’.

Food fortification is designed to combat the ‘hidden hunger’ problem by delivering foods rich in micronutrients to large populations, especially in developing countries. The plant at Isando can now produce the necessary vitamin A, B and mineral premixes to supplement basic foodstuffs and reduce the ‘hidden hunger’ problem. The plant is responsible for the supply of vitamins and nutritional mixes to its neighbouring countries in Africa.

AstraZeneca’s pharmaceutical production facility in Sixth of October City (Madinat Sittah Uktubar), near Cairo, produces a broad range of the company’s product portfolio including cardiovascular, psychiatric and cancer treatments. The plant was officially opened by Eyptian Health Minister Dr Hatem El-Gabaly on 11 December 2006.

AstraZeneca applied to the General Authority for Investment (GAFI) in Egypt for a license to build the plant in May 2004 and production began just a few months later in November. It was the firm’s first manufacturing investment in the Middle East, kicking off the company’s regional expansion strategy.

The new facility has 7,000m² of floor space and focuses on the three product lines. It cost $32m to construct and outfit.
“The new facility has 7,000m² of floor space and focuses on the three product lines.”

The initial products from the plant will be for sale in the Egyptian domestic market but further increases in production will supply nearby regions such as the European and Middle Eastern markets.

AstraZeneca hopes to gain a foothold in a potential expanding market which is worth an estimated $1.6bn annually. Up to 65% of the Egyptian market is supplied by multinational companies. Of this, 30% is through local manufacturing and 35% is through licensing agreements, according to AstraZeneca.

Employment at the AstraZeneca Eygpt Marketing Company more than doubled between 2005 and 2007 to a total 250 employees. It has now set up a regional office in Cairo responsible for the Middle East and North Africa.

AstraZeneca and Egyptian pharmaceutical market

Previously, AstraZeneca production in Egypt was carried out by a contract manufacturer under license and was only responsible for $12m of the company’s $18.8bn worldwide annual turnover.

The construction of the plant was helped by Eygpt’s adoption of the WTO ‘TRIPS’ agreement from 1 January 2005, which protects intellectual property rights.

Egypt is the largest producer and consumer of pharmaceuticals in the Middle East and North Africa (MENA) region with a 30% share of the supply in these markets. The region also absorbs most of Egypt’s pharmaceuticals exports. Egypt surely has a vested interest in attracting big pharmaceutical companies so that it can become a production hub for the future of the Middle Eastern and African markets.

AstraZeneca has identified Egypt as one of the key emerging markets for further development, together with such countries as China and Mexico.

Contractor and facility

The general construction contract was awarded to Orascom Construction Industries (OCI) of Egypt, who carried out construction of the main production building, support facilities, administration building, laboratories, packing, dispatching and warehouse facilities.

The internal design and outfitting contract was awarded to Uhde and Chemgineering. These two companies, based in Germany and Switzerland respectively, are responsible for the detailed design and planning of the production facility including definition of the process equipment (tablet and capsule production and packaging), cleanroom facilities, GMP concepts, master planning and technology transfer.

The Chemgineering / Uhde consortium also coordinated with a local pharmaceutical engineering / design contractor, Sabbour Associates, who was responsible for elements of plant design and construction and project management. Sabbour Associates have been the main contractor on two other pharmaceutical facilities for both Sedico and October Pharma in Sixth of October City and have plenty of experience of the region.

“The high-speed tablet presses are capable of producing up to 12,000 tablets per minute.”

The facility initally has a capacity of 250 million tablets per year, but the production lines and the attendant compounding, pressing, drying, packaging, warehousing and other production infrastructure will be arranged so that a future expansion (which is already on the drawing board) can allow the production of in excess of 400 million tablets per year.

Tableting process

The facility uses the latest state-of-the-art processes to produce solid dosage forms (tablets, hard and soft gelatine capsules and a variety of delayed release forms). The tablets will be produced where possible by wet / dry granulation methods, but also by direct compression if necessary.

The processes will allow for the production of extended and delayed release dosage forms of various designs and also micro particle coated capsules for incorporation in hard gelatine capsules.

The high-speed tablet presses are capable of producing up to 12,000 tablets per minute, but the average tablet press speed is 3,000 tablets per minute. Press speed requires powders / granules to have good fluid properties (product flowability) – if they do not the formulation needs to be modified by using additives (excipients) or the press speed may need to be slower.

The inauguration ceremony of the new manufacturing plant was attended by Hailemariam Desalegn, the Prime Minister of Ethiopia.

The plant is expected to become fully operational in the second quarter of 2013. The investment for the construction of the plant was about ETB170m ($9.17m).

Details of Julphar’s pharmaceutical facility in Ethiopia

The new facility has a total floor space of about 40,000ft2. It will produce solid as well as liquid dosage forms of medicines. It will manufacture about 25 million bottles of suspension and syrup, 500 million tablets, and more than 200 million capsules.

It will provide many employment opportunities and is expected to recruit 50 local staff for various positions such as quality assurance, quality control, production and maintenance, regulatory affairs, and administration services.

The new plant was built in order to produce relevant and much needed medicines for Ethiopian market. The pharmaceutical products produced at the new facility will be exported throughout Africa. Medtech manages the sales and marketing of the products and will become a key supplier in the local as well as other markets in the African continent.

Technology and products manufactured at Julphar Addis Ababa plant

The new manufacturing facility is equipped with advanced technology processes to produce high-quality pharmaceutical products for use in the domestic market and other African countries.

Pharmaceutical products developed by Julphar include antibiotic, biotech, cardiovascular, dermatology, insulin, metabolic, over the counter, pulmonary, women’s health, and paramedical solutions.

The facility also has a pharmaceuticals packaging line equipped with labelling, cartoning, shrink wrapping, packing, and blistering machines.

Construction of Julphar’s new pharma facility in Ethiopia

The new plant was designed as well constructed by Julphar’s engineering and construction division. It was constructed with cGMP-compliance. Julphar is also working towards obtaining international accreditation for the plant.

The African pharmaceuticals market

“Africa’s healthcare market is growing at an annual rate of 10.6%.”

The African pharmaceuticals market has been growing at a fast pace. The continent has a population close to a billion. The pharmaceutical market in Africa is set to grow between $8bn and $10bn a year, with pharmaceutical spending in Africa expected to increase to about $30bn by 2016.

Africa’s healthcare market is growing at an annual rate of 10.6%. Industry growth is attributed to the extensive surge of middle-class spending on diseases, and rapid urbanisation. The industry has been growing fast despite the infrastructural shortcomings in many of the African countries.

Julphar company information

Julphar is a leading pharmaceutical company in the UAE and was established in 1980. It is involved in the production and distribution of pharmaceutical products across 40 countries worldwide. It has more than 800 product offerings in various dosage forms in the pipeline.

The new facility at Addis Ababa was constructed as part of Julphar’s international expansion strategy. Julphar aims to play a major role in the pharmaceuticals industry in future through the expansion. It currently has 11 plants in Ras Al Khaimah, UAE.