apceth Biopharma GmbH manufactures DCprime’s cell-based cancer vaccine for phase II clinical study

Munich, Germany, November 16, 2018. apceth Biopharma GmbH, an established contract manufacturing organization in the field of gene and cell therapy, has started supplying clinical batches of DCprime’s cell-based cancer vaccine DCP-001 to a phase II clinical study for the treatment of Acute Myeloid Leukemia (AML). AML is a haematological cancer characterized by high risk of relapse, even after initial response to chemotherapy. Cancer vaccination with dendritic cells could be a successful strategy to boost the patient’s immune system and result in lasting disease control. DCprime announced yesterday that the first patient has been treated with the product DCP-001 at the Amsterdam University Medical Center (UMC). “We are proud to be the manufacturing partner of DCprime for DCP-001, the first off-the shelf dendritic cell vaccine in a cancer indication with high unmet medical need”, explained Dr Christine Günther, CEO of apceth Biopharma. “Our long-term collaboration with DCprime has always been constructive and cooperative, from technology transfer to the large-scale manufacturing process.” Dr Erik Manting, CEO of DCprime, commented: “Based on the recently published encouraging results of the phase I trial with our lead product DCP-001, we aim to confirm these results in the phase II ADVANCE-II trial and we are happy with the enrollment of the first patient. We would like to thank the apceth Biopharma team for their important contribution to the manufacturing of our product.” About apceth Biopharma GmbH apceth Biopharma is a pioneering company in cell therapies and regenerative medicine with an innovative portfolio of drug candidates for the treatment of inflammation, autoimmunity and solid tumors. apceth is developing two proprietary next generation gene therapy products, which are based on the introduction of therapeutic transgenes into mesenchymal stem cells (MSCs). apceth-201 expresses the immunomodulatory protein alpha-1 antitrypsin for the treatment of graft-versus-host disease. The company’s second program, apceth-301, expresses a potent immunostimulatory “cocktail” of cytokines, which locally activates the immune system to eradicate tumor cells. apceth-301 is currently being developed for glioblastoma as well as for other solid tumors. apceth Biopharma is also a leading and certified Contract Development and Manufacturing Organization for cell and gene therapeutics with a broad international customer base. apceth owns state-of-the-art manufacturing facilities with Grade B/A, C and D cleanrooms (ISO 5, ISO 7, ISO 8, BSL2) and is certified according to regulatory requirements for cell and gene therapies (Advanced Therapy Medicinal Products, ATMPs). The company has a comprehensive expertise in GMP manufacturing of autologous and allogeneic cell types that are either native or genetically modified. It has long-standing experience with various cell products, including mesenchymal stem cells (MSCs), hematopoietic stem cells (HSC), lymphocytes, monocytes, dendritic cells, cord blood derived stem cells, and has the potential to expand to CAR-T and induced pluripotent stem cell (iPSC) technologies. apceth has successfully obtained manufacturing licenses for multiple cell therapy products for clinical as well as commercial use. apceth’s CDMO team provides fully customized solutions in the development and production of every customer product and process. Located centrally in the heart of Europe, apceth can perform efficient and fast supply for patients all over the continent. Press release as pdf (English) Press release as pdf (German) Contact apceth Biopharma GmbH Dr Christine Guenther, CEO Max-Lebsche-Platz 30 81377 Munich Germany Phone: +49 (0)89 7009608 0 Email: contact@apceth.com www.apceth.com

apceth Biopharma presents positive data demonstrating efficacy of apceth-201 in preclinical models of acute GvHD

Munich, Germany, Oct 19, 2018 – apceth Biopharma GmbH, a company with the mission of improving patients’ lives with next generation cell therapies, announced today positive results for apceth-201 in mouse models for acute graft-versus-host disease (aGvHD). The data was highlighted in a poster presentation at the Annual Congress of the European Society of Gene and Cell Therapy in Lausanne on October 16-19. “We are very excited with these results that clearly show the transformative therapeutic potential of apceth-201 in an immune-mediated disease with high unmet medical need and few treatment options”, said Dr Christine Guenther, CEO of apceth Biopharma. “Based on these promising and impressive data, we are currently preparing to initiate a phase 1/2 clinical study assessing the safety and efficacy of apceth-201 in adult steroid-refractory GvHD patients.” Acute graft-versus-host disease is a frequent complication associated with allogeneic hematopoietic stem cell transplantation (HSCT). Immunosuppressants are used to manage aGvHD; however, steroid-refractory aGvHD develops in many cases and has an extremely poor prognosis. apceth-201 significantly improved clinical score and overall survival in two aGvHD models apceth-201 are allogeneic human mesenchymal stromal cells (MSCs) that have been genetically modified to express alpha-1 antitrypsin (AAT), a protease inhibitor exerting potent anti-inflammatory and tissue-protective functions to further augment the immunomodulatory potential of MSCs. In vitro assays demonstrated that apceth-201 efficiently suppresses T-cell proliferation, the maturation of dendritic cells, and also polarizes macrophages to an anti-inflammatory M2 type. The in vivo efficacy of apceth-201 was assessed in two different mouse models for aGvHD. In a humanized model vehicle-treated control animals succumbed quickly to GvHD, whereas median survival was doubled in apceth-201-treated animals. Mice receiving apceth-201 showed significantly improved clinical scores, a striking amelioration of bone marrow cellularity, and reduced levels of inflammatory markers. apceth-201 was further tested in a GvHD model system which closely mimics haploidentical HSCT. Vehicle-treated control animals again succumbed quickly to GvHD, whereas treatment with apceth-201 resulted in long-term survival of 57 % of the mice. Initially, all apceth-201-treated animals exhibited clinical scores comparable to the control animals. However, within a period of 25 days after the second cell injection, the clinical scores had returned to baseline, indicating complete resolution of GvHD. This promising data has led to planning of a phase 1/2 study using apceth-201 for the treatment of steroid-refractory aGvHD in adults.   Press release as pdf   About apceth Biopharma GmbH apceth Biopharma is a pioneering company in cell therapies and regenerative medicine with an innovative portfolio of drug candidates for the treatment of inflammation, autoimmunity and solid tumors. apceth is developing two proprietary next generation gene therapy products, which are based on the introduction of therapeutic transgenes into mesenchymal stem cells (MSCs). apceth-201 expresses the immunomodulatory protein alpha-1 antitrypsin for the treatment of graft-versus-host disease. The company’s second program, apceth-301, expresses a potent immunostimulatory “cocktail” of cytokines, which locally activates the immune system to eradicate tumor cells. apceth-301 is currently being developed for glioblastoma as well as for other solid tumors. apceth Biopharma is also a leading and certified Contract Development and Manufacturing Organization for cell and gene therapeutics with a broad international customer base. apceth owns state-of-the-art manufacturing facilities with Grade B/A, C and D cleanrooms (ISO 5, ISO 7, ISO 8, BSL2) and is certified according to regulatory requirements for cell and gene therapies (Advanced Therapy Medicinal Products, ATMPs). The company has a comprehensive expertise in GMP manufacturing of autologous and allogeneic cell types that are either native or genetically modified. It has long-standing experience with various cell products, including mesenchymal stem cells (MSCs), hematopoietic stem cells (HSC), lymphocytes, monocytes, dendritic cells, cord blood derived stem cells, and has the potential to expand to CAR-T and induced pluripotent stem cell (iPSC) technologies. apceth has successfully obtained manufacturing licenses for multiple cell therapy products for clinical as well as commercial use. apceth’s CDMO team provides fully customized solutions in the development and production of every customer product and process. Located centrally in the heart of Europe, apceth can perform efficient and fast supply for patients all over the continent. Contact apceth Biopharma GmbH Dr Christine Guenther, CEO Max-Lebsche-Platz 30 81377 Munich Germany Phone: +49 (0)89 7009608 0 Email: contact@apceth.com www.apceth.com

BIO-Europe 2018

Copenhagen, Denmark, November 5–7, 2018. apceth Biopharma booth #72 – Europe’s largest life science partnering conference – conference website BIO-Europe’s world-class workshops, panels and active exhibition along with thousands of prescheduled one-to-one meetings make this event an unrivaled forum for companies across the biotech value chain to meet and do business. (Citation from conference website)

apceth to present at European Society of Gene and Cell Therapy Annual Congress

Munich, Germany, Oct 10, 2018. apceth Biopharma is proud to announce that it will be presenting its latest results on apceth-201 in graft-vs-host-disease (GvHD) at this year’s ESGCT Annual Congress “Changing Modern Medicine: Stem Cells & Gene Therapy”. The meeting will take place in Lausanne, Switzerland, on October 16-19, 2018. apceth‘s poster is titled: “Human Mesenchymal Stem Cells Genetically Engineered to Express Alpha-1 Anti-Trypsin (apceth-201) Confer a Long-Term Survival Benefit in Lethal Mouse Models of Graft-Versus-Host-Disease”. It has poster number P126 and will be presented during the session on Thursday, October 18. For further information on ESGCT Annual Congress, please visit https://www.esgct.eu/Congress/Current-Congress.aspx

apceth to manufacture first potential one-time gene therapy for transfusion dependent β-thalassemia

Munich, Germany, Oct 5th, 2018 – apceth Biopharma GmbH, a leading company for the development and manufacturing of cell and gene therapeutics, congratulates its partner bluebird bio on the acceptance and validation of its market authorization application (MAA) by the European Medicines Agency (EMA). bluebird bio has applied for market authorization of its investigational LentiGlobin™ gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype. “We congratulate our partner bluebird bio on this recent success”, commented Dr Christine Guenther, CEO of apceth Biopharma. “These are exciting times also for apceth, as we are now in the position to become one of the very few companies worldwide to manufacture a cell-based gene therapy for commercial use.” apceth has supported and will continue to support bluebird bio for obtaining the market authorization for LentiGlobin™. Following a successful long-term manufacturing relationship, apceth Biopharma and bluebird bio entered a commercial drug product manufacturing agreement in 2016. apceth Biopharma is bluebird bio’s clinical and commercial manufacturing partner for Europe not only for LentiGlobin, but also for bluebird bio’s product candidate Lenti-D for cerebral adrenoleukodystrophy.   Press release as pdf (English)  or pdf (German) Press release from bluebird bio     About apceth Biopharma GmbH apceth Biopharma is a pioneering company in cell therapies and regenerative medicine with an innovative portfolio of drug candidates for the treatment of inflammation, autoimmunity and solid tumors. apceth is developing two proprietary next generation gene therapy products, which are based on the introduction of therapeutic transgenes into mesenchymal stem cells (MSCs). apceth-201 expresses the immunomodulatory protein alpha-1 antitrypsin for the treatment of graft-versus-host disease. The company’s second program, apceth-301, expresses a potent immunostimulatory “cocktail” of cytokines, which locally activates the immune system to eradicate tumor cells. apceth-301 is currently being developed for glioblastoma as well as for other solid tumors. apceth Biopharma is also a leading and certified Contract Development and Manufacturing Organization for cell and gene therapeutics with a broad international customer base. apceth owns state-of-the-art manufacturing facilities with Grade B/A, C and D cleanrooms (ISO 5, ISO 7, ISO 8, BSL2) and is certified according to regulatory requirements for cell and gene therapies (Advanced Therapy Medicinal Products, ATMPs). The company has a comprehensive expertise in GMP manufacturing of autologous and allogeneic cell types that are either native or genetically modified. It has long-standing experience with various cell products, including mesenchymal stem cells (MSC), hematopoietic stem cells (HSC), lymphocytes, monocytes, dendritic cells, cord blood derived stem cells, and has the potential to expand to CAR-T and induced pluripotent stem cell (iPSCs) technologies. apceth has successfully obtained manufacturing licenses for multiple cell therapy products for clinical as well as commercial use. apceth’s CDMO team provides fully customized solutions in the development and production of every customer product and process. Located centrally in the heart of Europe, apceth can perform efficient and fast supply for patients all over the continent.   Contact apceth Biopharma GmbH Dr Christine Guenther, CEO Max-Lebsche-Platz 30 81377 Munich Germany   Phone: +49 (0)89 7009608 0 Email: contact@apceth.com www.apceth.com

apceth Biopharma receives an extended general manufacturing authorization for cell and gene therapeutics

Munich, Germany, Aug 7, 2018 – apceth Biopharma GmbH, a leading company for the development and manufacturing of cell and gene therapeutics, announced today that it has received an extended general manufacturing authorization by the District Government of Upper Bavaria. This manufacturing authorization for cell and gene therapeutics is now regulated on an activity-related and no longer product-related basis. The previously obligatory inspection by the regulatory authority becomes optional. “We are very pleased with the trust and confidence shown by the regulatory authority”, commented Dr Christine Guenther, CEO of apceth Biopharma. “I am very proud of the entire apceth team, which has successfully completed every inspection by the District Government of Upper Bavaria and the Paul-Ehrlich-Institute since the foundation of the company. This kind of regulatory approval is rarely found for cell and gene therapeutics.” The extended general manufacturing authorization includes the clinical as well as commercial supply of cell products with different manufacturing techniques. It allows the production of cell therapeutics from various starting materials, for example bone marrow and umbilical cord blood, the production of native and genetically modified cells as well as the autologous or allogeneic administration. “This gives us more flexibility, streamlines the manufacturing approval process and shortens the timelines for our new customers”, explains Dr Andreas Schmiede, head of the Contract Development and Manufacturing department (CDMO). In 2018, apceth has also expanded its manufacturing capacities with two new class B clean rooms and is continuously strengthening its Manufacturing and Quality Control teams. “Due to the high demand for capacity for the CDMO business, we are significantly enhancing our capabilities to meet the growing need of our customers”.   About apceth Biopharma GmbH apceth Biopharma is a pioneering company in cell therapies and regenerative medicine with an innovative portfolio of drug candidates for the treatment of inflammation, autoimmunity and solid tumors. apceth is developing two proprietary next generation gene therapy products, which are based on the introduction of therapeutic transgenes into mesenchymal stem cells (MSCs). apceth-201 expresses the immunomodulatory protein alpha-1 antitrypsin for the treatment of graft-versus-host disease. The company’s second program, apceth-301, expresses a potent immunostimulatory “cocktail” of cytokines, which locally activates the immune system to eradicate tumor cells. apceth-301 is currently being developed for glioblastoma as well as for other solid tumors. apceth Biopharma is also a leading and certified Contract Development and Manufacturing Organization for cell and gene therapeutics with a broad international customer base. apceth owns state-of-the-art manufacturing facilities (ISO 5, ISO 7, ISO 8, BSL2 cleanrooms) and is certified according to regulatory requirements for cell and gene therapies (Advanced Therapy Medicinal Products, ATMPs). The company has a comprehensive expertise in GMP manufacturing of autologous and allogeneic cell types that are either native or genetically modified. It has long-standing experience with various cell products, including mesenchymal stem cells (MSC), hematopoietic stem cells (HSC), lymphocytes, monocytes, dendritic cells, cord blood derived stem cells, and has the potential to expand to CAR-T and induced pluripotent stem cell (iPSCs) technologies. apceth has successfully obtained manufacturing licenses for multiple cell therapy products for clinical as well as commercial use. apceth’s CDMO team provides fully customized solutions in the development and production of every customer product and process. Located centrally in the heart of Europe, apceth can perform efficient and fast supply for patients all over the continent.   Contact apceth Biopharma GmbH Dr Christine Guenther, CEO Max-Lebsche-Platz 30 81377 Munich Germany Phone: +49 (0)89 7009608 0 Email: contact@apceth.com www.apceth.com Download the  press release (pdf, English or German)

Senior Quality Control Manager (m/w)

Zur Verstärkung unseres Teams am Standort Ottobrunn suchen wir einen Senior Quality Control Manager (m/w) Bitte senden Sie uns Ihre vollständigen Bewerbungsunterlagen mit Angabe der Projekt-Nummer, des möglichen Eintrittstermins und Ihrer Gehaltsvorstellung per Email an career@apceth.com.

Quality Control Manager (m/w)

Zur Verstärkung unserer Abteilung Quality Control am Standort Ottobrunn suchen wir einen Quality Control Manager (m/w) Bitte senden Sie uns Ihre vollständigen Bewerbungsunterlagen mit Angabe der Projekt-Nummer, des möglichen Eintrittstermins und Ihrer Gehaltsvorstellung per Email an career@apceth.com.

Prozess-Ingenieur (m/w)

Aufgrund der weiteren Expansion unseres Unternehmens suchen wir zur Verstärkung unseres Teams am Standort Ottobrunn zum nächstmöglichen Zeitpunkt einen Prozess-Ingenieur (m/w) Bitte senden Sie uns Ihre vollständigen Bewerbungsunterlagen mit Angabe der Projekt-Nummer, des möglichen Eintrittstermins und Ihrer Gehaltsvorstellung per Email an career@apceth.com.

2018 BIO International Convention

Location: Boston, MA 04.06.2018 – 07.06.2018 Conference Webpage “The BIO International Convention is hosted by the Biotechnology Innovation Organization (BIO), which represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations.” (Citation: Conference Webpage)