Merck Millipore Adds Protein Pegylation to Portfolio of Services Through Collaboration

Merck Millipore recently announced a collaboration with celares GmbH to provide pegylation services to customers developing protein-based therapeutics and biosimilars. The new service offering enabled by the collaboration includes feasibility studies, process and analytical development, and scale-up from milligram-to-gram quantities required for pilot and subsequent commercial scale.

Pegylation can significantly improve the pharmacological and physicochemical properties of peptide and protein therapeutics and reduce side effects. This well-recognized delivery system for biologics can enhance protein stability, bioavailability, and solubility, overcoming common challenges in the development of these therapeutics.

“Whilst a widely used approach to improving drug delivery, a successful pegylation strategy is dependent upon materials expertise and application experience,” said
Andrew Bulpin, Executive Vice President of Process Solutions, Merck Millipore. “celares is a recognized leader in this area and through our collaboration, we are now able to expand our service offering to include conjugation, further helping our biopharmaceutical and biosimiliar customers to optimize their protein therapeutics and to reduce their time to market.”

The new services will leverage EMD Millipore’s broad range of functionalized PEG products of different molecular weight and activation chemistry, as well as buffers,
solvents, and excipients, unit operations employed during the pegylation process, and subsequent purification, including tangential and normal flow filtration and
chromatography.

“An optimized formulation can help a promising protein therapeutic reach the clinic and patients in need,” said Dr. Frank Leenders, Managing Director of Operations at celares GmbH. “Our expertise in pegylation combined with the experience and integrated products and unit operations from EMD Millipore will create customized solutions needed by the biopharmaceutical industry to maintain forward progress of promising drug candidates.”

Merck Millipore is the Life Science subsidiary of Merck, Darmstadt, Germany. As part of the global Life Science business of Merck, Merck Millipore offers a broad range of innovative performance products, services, and business relationships that enable its customers’ success in research, development, and production of biotech and pharmaceutical drug therapies. For more information, visit www.merckmillipore.com.

Abeona Therapeutics Announces License for Next-Generation AAV Delivery Vector
Abeona Therapeutics, Inc. recently announced a license agreement with Stanford University for an AAV (adeno-associated virus) delivery vector for the treatment of Fanconi anemia (FA) and rare blood disease platform. This license augments a previously announced license agreement with the University of Minnesota for ABO-301 (AAV-FANCC) to treat patients with Fanconi anemia (FA) disorder and other rare blood diseases.

“Effective product development in gene therapy requires choosing the right delivery vehicle because each virus has a unique transduction profile that determines its ability to transfer genetic material from one organism to another,” said Steven Rouhandeh, Executive Chairman. “This licensing transaction complements our near-clinical stage programs in Sanfilippo syndromes (MPS IIIA and MPS IIIB), and juvenile Batten disease, as well as further expands our commitment to building a leadership position in the gene therapy space, with a focus on developing therapies for patients with rare diseases.”

The major function of bone marrow is to produce new blood cells. In FA, a DNA mutation renders the FANCC gene nonfunctional. Loss of FANCC causes patient skeletal abnormalities and leads to bone marrow failure. Fanconi anemia patients also have much higher rates of hematological diseases, such as acute myeloid leukemia (AML) or tumors of the head, neck, skin, gastrointestinal system, or genital tract. The likelihood of developing one of these cancers in people with Fanconi anemia is between 10% and 30%. Aside from bone marrow transplantation (BMT), there are no specific treatments known that can halt or reverse the symptoms of FA. Repairing fibroblast cells in FA patients with a functional FANCC gene is the focus of our AAV-based gene therapy approach.

“The FANCC gene encodes a protein that functions as part of the pathway responsible for genome surveillance and repair of DNA damage. Hematopoietic progenitors are an ideal population for correction for therapeutic use. Our strategies encompass first- and second-generation therapies to correct hematopoietic cells,” added Tim Miller, PhD, President & CEO. “We are leveraging the unique capabilities of the CRISPR-Cas9 gene editing platform technology to build a robust product pipeline to address the unmet needs of patients that may have one of a variety of blood diseases, which include Beta-thalassemia, Fabry disease, as well as inherited forms of neutropenia, thrombocytopenia, and anemias.”

ABO-301 is an AAV-based gene therapy that has shown promising preclinical efficacy in delivery of a normal copy of the defective gene to cells of the hematopoietic or blood system with the aim of reversing the effects of the genetic errors that cause Fanconi anemia. Using a novel CRISPR (clustered, regularly interspaced short palindromic repeats)-Cas9 (CRISPR associated protein 9) system, researchers used a protein-RNA complex composed of an enzyme known as Cas9 bound to a guide RNA molecule that has been designed to recognize a particular DNA sequence. The RNA molecules guide the Cas9 complex to the location in the genome that requires repair. CRISPR-Cas9 uniquely enables surgically efficient knock-out, knock-down or selective editing of defective genes in the context of their natural promoters, unlocking the potential to treat both recessive and dominant forms of genetic diseases. Most importantly, this approach has the potential to allow safer, more precise gene modification for a wide range of rare blood diseases.

Abeona Therapeutics, Inc. develops and delivers gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona’s lead programs are AB0-101 (AAV9 NAGLU) and ABO-102 (scAAV9 SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA) in collaboration with patient advocate groups, researchers and clinicians, anticipated to commence clinical trials in 2015. The company is also developing ABO-201 (scAAV9 CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, it is also developing rare plasma protein therapies, including SDF AlphaT (alpha-1 protease inhibitor) for inherited COPD using its proprietary SDFT (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com.