We staan aan de start van een nieuwe groene revolutie, dankzij een groep Amerikaanse wetenschappers. Door aanpassingen in het DNA van de plant lukte het hen om de groei van planten met 40% te laten toenemen. Dat is een belangrijke doorbraak voor de mondiale voedselproductie. De vraag naar voedsel zal de komende decennia namelijk fors toenemen door een groeiende wereldbevolking, terwijl het areaal landbouwgrond door de klimaatverandering juist afneemt.
Planten zijn in staat om via een ingenieus biologisch proces, de fotosynthese, uit zonlicht en koolstofdioxide (CO2) hun eigen voedingsstoffen (suikers) te maken. Tijdens de fotosynthese komen er naast de nuttige producten zoals suikers en zuurstof (O2), ook voor de plant schadelijke afvalstoffen vrij. Het opruimen van deze schadelijke producten kost de plant veel energie, die ze niet voor haar groei in kan zetten. Door met behulp van genetische modificatie een veel efficiëntere route voor de afbraak van deze afvalstoffen te maken, nam de groei van de plant tot wel 40% toe. De volgende stap is om die groeipotentie gericht te bevorderen in de ‘nuttige’ delen van de plant, bijvoorbeeld de vruchten, zaden, bladeren of stengels. Het bereiken van dit resultaat zal nog wel een aantal jaren duren, net als de toelating van de planten tot de markt. In Europa geldt namelijk een zeer streng, langdurig en onzeker traject voor genetisch gemodificeerde gewassen. HollandBIO pleit al jaren voor aanpassing van deze wetgeving. Deze doorbraak bevestigt maar weer eens hoe belangrijk de modernisering van het biotech beleid is, willen de EU en Nederland straks de vruchten van deze mooie vindingen kunnen plukken.
Galapagos expands its clinical study program with GLPG1690 in systemic sclerosis, following the recent start of the ISABELA Phase 3 program with ‘1690 in IPF.
NOVESA is a double-blind, placebo-controlled Phase 2a trial evaluating the efficacy, safety and PK/PD of ‘1690 in patients with systemic sclerosis (SSc, or scleroderma). NOVESA is planned to recruit 30 patients with diffuse cutaneous SSc, an autoimmune disease involving multiorgan fibrosis, which has one of the highest mortality rates among rheumatic diseases1 . One of the most visible manifestions is hardening of the skin. In diffuse cutaneous SSc, skin thickening affects several body areas, and patients have a higher risk of developing fibrosis of various internal organs, such as the lung. Currently, there are no approved drugs for this disease. SSc affects approximately 90,000 patients in the US and Europe, with a predominance of female patients (75%).
The primary endpoint of NOVESA is the modified Rodnan skin score (mRSS) at 24 weeks. mRRS measures the skin thickness as a surrogate measure of disease severity and mortality, with an increase in thickness associated with involvement of internal organs and increased mortality2 . Secondary objectives and exploratory endpoints include FVC3 , HRCT4 , quality of life as measured by QoL-Q (SHAQ)5 , and CRISS6 , a SSc disease composite score.
“In addition to our Phase 3 program in IPF7 , we are excited to broaden our development program with ‘1690 to a second indication,” said Dr. Walid Abi-Saab, Chief Medical Officer at Galapagos. “Moreover, SSc is particularly interesting, as this disease straddles our expertise in autoimmune diseases as well as in fibrosis. Thanks to the broad MoA of ‘1690, which is both anti-inflammatory and anti-fibrotic, this compound has the potential to address the important unmet medical need in SSc.”
Fibrocor Therapeutics, a privately held Canadian company, and Galapagos announced a global partnership focused on a novel target for idiopathic pulmonary fibrosis (IPF) and other indications.
Fibrocor specializes in the development of tissue-specific therapeutics to treat the underlying cause of fibrotic diseases of the kidney and other organs. The collaboration announced today concerns a small molecule inhibitor program, currently in the lead optimization stage of development for the treatment of fibrotic diseases of the lung and other organs, the target of which is undisclosed. In exchange for global commercialization rights to Galapagos, Fibrocor will receive an upfront payment, and potentially is eligible for further milestone and royalty payments. Galapagos will be responsible for all further development of the program.
“This collaboration validates the fibrosis drug development expertise of Fibrocor,” says Mark A. Steedman, President and CEO of Fibrocor. “I take my hat off to Dr. Richard Gilbert and the scientific team, including Evotec GmbH, our CRO1 partner, for establishing a compelling data package that ultimately attracted Galapagos, a world-renowned biotech company with a key franchise in fibrosis. We feel this is the beginning of a great relationship and look forward to working with Galapagos to the benefit of fibrosis sufferers everywhere.”
“The collaboration with Fibrocor announced today is an excellent strategic fit for Galapagos, as we continue to expand our franchise in IPF, and more broadly, in fibrosis,” says Dr. Piet Wigerinck, Chief Scientific Officer at Galapagos. “We are enthusiastic about the drug discovery approach at Fibrocor, and look forward to collaborating with the team to address the large unmet medical need in fibrosis.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-01-04 11:30:002019-01-08 22:14:40Fibrocor and Galapagos sign partnership in fibrosis← #nieuws
HollandBIO, de belangenvereniging voor biotech bedrijven in Nederland, heeft een duidelijke stip aan de horizon: een samenleving waarin biotech innovaties maximaal bijdragen aan onze gezondheid, een duurzame samenleving en een bloeiende economie.
HollandBIO draagt bij aan het succes van de Nederlandse life sciences sector door bedrijven te vertegenwoordigen en te ondersteunen. We zijn een snelgroeiende vereniging. Een ambitieus en enthousiast team (9 FTE) is verantwoordelijk voor de activiteiten van HollandBIO.
De functie
HollandBIO is op zoek naar een multi-tasker, die zorgt dat alles op rolletjes loopt. Je bent het eerste aanspreekpunt voor externen en de stabiele factor voor het team. Je bent de sparring-partner van de Managing Director en ondersteunt het hele team. Je rapporteert aan de Managing Director.
Secretariële ondersteuning
Je handelt inkomende telefoontjes en mailtjes zelfstandig af
Voor het teamoverleg maak je de agenda en houd je de actiepunten bij
Je beheert de agenda van de Managing Director
Je boekt (buitenlandse) reizen en overnachtingen
Administratie
Je houdt het CRM en de ledenadministratie up tot date
Je bewaakt de tijdslijnen van interne administratieve processen
Evenementen
Je plant, organiseert en coördineert de evenementen
Office management
Je houdt de voorraad kantoorbenodigdheden bij en bestelt wanneer nodig
Je bent contactpersoon voor leveranciers en onderhoud
Jouw profiel
Jij ziet het werk liggen en hebt de dingen al gedaan voordat ze worden gevraagd. Je kunt snel schakelen tussen uiteenlopende activiteiten, hebt een gezonde dosis lef en weet prioriteiten te stellen. Je maakt er een persoonlijke missie van om de perfecte locatie te vinden voor onze events en bijeenkomsten. Je gaat zelfstandig te werk en durft initiatief te tonen. Kortom: we zoeken iemand die alles kan en ook nog eens stressbestendig en gezellig is!
Een MBO+ / HBO werk- en denk niveau, minimaal 3 jaar ervaring in een vergelijkbare functie
Een goede beheersing van het Nederlands en Engels
Uitstekende Microsoft Office skills
Organisatietalent, ervaring met de organisatie van evenementen is een pré
Discreet, zorgvuldig en organisatiesensitief
Representatief en je communiceert makkelijk en natuurlijk met iedereen
Ons aanbod
Positieve en energieke werkomgeving
Goed arbeidsvoorwaardenpakket
Werken in een klein, professioneel team
Geïnteresseerd?
Stuur voor 15 januari 2019 je motivatie en CV t.a.v. Annemiek Verkamman (Managing Director) naar info@hollandbio.nl. De eerste gesprekken zijn op dinsdag 22 januari en woensdag 23 januari. Voor meer informatie over de functie neem je contact op met Nicole Schönherr (Management Assistent), via telefoonnummer 070-8331333.
Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, has initiated a Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9 AAV9-CLN3 (AAV9-CLN3) gene therapy in children with CLN3 Batten disease. Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. The initial patient completed a one-month observation period following dosing with no serious adverse events reported to date.
This first in human study of an investigational gene therapy in CLN3 Batten disease, a life-threatening genetic neurologic disorder that typically begins in early childhood and results in premature death, is currently being conducted at Nationwide Children’s Hospital (Columbus, Ohio). CLN3 is the most prevalent of the Batten’s disorders affecting an estimated 5,000+ patients.
“We are pleased to announce that the first child has been dosed in the Phase 1/2 study for CLN3 Batten disease,” said Jay Barth, MD, Chief Medical Officer at Amicus Therapeutics, Inc.“With this pioneering study in CLN3 Batten disease, a severe and devastating neurodegenerative disorder with no approved treatments, we hope that a single intrathecal administration of our gene therapy has the potential to treat the underlying cause of disease and provide a durable and meaningful treatment benefit. This initial participant in our CLN3 Batten disease study, together with the participants in our ongoing clinical study in CLN6 Batten disease, further supports the encouraging safety profile for the intrathecal AAV delivery platform. Advancing this program is another important step forward to potentially improve the lives of thousands of children living with Batten disease and other neurologic lysosomal storage disorders.”
Emily C. De Los Reyes, MD, Principal Investigator at Nationwide Children’s stated, “The initiation of this clinical study in CLN3 Batten disease is an important initial milestone. The proof-of-concept demonstrated in preclinical studies and validation of the intrathecal AAV platform across multiple indications here at Nationwide Children’s Hospital provides promise for the delivery approach in this study. We look forward to enrolling additional participants in the study and comparing the findings to the existing natural history data in CLN3 Batten disease.”
The Phase 1/2 study is enrolling children aged 3 to 10 years with a confirmed diagnosis of CLN3 Batten disease in two sequential intrathecal dose groups: a one-time low-dose of AAV9-CLN3 (Group 1) and a one-time high-dose of AAV9-CLN3 (Group 2) after evaluation of Group 1 participants. Both groups will participate in the current study for a period of three years. Amicus plans to present clinical data from this study, including interim data, at future scientific congresses and other relevant venues. The primary outcome measures are safety and efficacy as determined using the physical disability subscale of the Unified Batten Disease Rating Scale (UBDRS) in CLN3 Batten disease. More information is available at www.clinicaltrials.gov: NCT03770572.
CLN3 Batten disease, the most common form of NCL results from a mutation in the CLN3 gene which primarily affects the nervous system. Children with this condition develop vision impairment, intellectual disability, progressive loss of motor function, speech difficulties, and seizures which worsen over time.
ProQR has received a Fast Track designation from the Food and Drug Administration (FDA) for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Fast Track designation is granted by FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.
“We are very pleased with the Fast Track designation the FDA granted us for QR-421a. Patients with Usher syndrome, the leading cause of combined deafness and blindness, currently have no available therapies for their vision loss and this designation emphasizes the high unmet need in this disease,” said Daniel de Boer, Chief Executive Officer of ProQR. “We are also looking forward to begin enrollment in the Phase 1/2 STELLAR clinical trial in the coming months with preliminary data expected in mid-2019.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-01-03 21:32:412019-01-08 22:15:51ProQR Receives Fast Track Designation from FDA for QR-421a for Usher Syndrome Type 2← #nieuws
Bristol-Myers Squibb and Celgene have entered into a definitive merger agreement under which Bristol-Myers Squibb will acquire Celgene in a cash and stock transaction with an equity value of approximately $74 billion.
Under the terms of the agreement, Celgene shareholders will receive 1.0 Bristol-Myers Squibb share and $50.00 in cash for each share of Celgene. Celgene shareholders will also receive one tradeable Contingent Value Right (CVR) for each share of Celgene, which will entitle the holder to receive a payment for the achievement of future regulatory milestones. The Boards of Directors of both companies have approved the combination.
The transaction will create a leading focused specialty biopharma company well positioned to address the needs of patients with cancer, inflammatory and immunologic disease and cardiovascular disease through high-value innovative medicines and leading scientific capabilities. With complementary areas of focus, the combined company will operate with global reach and scale, maintaining the speed and agility that is core to each company’s strategic approach.
Based on the closing price of Bristol-Myers Squibb stock of $52.43 on January 2, 2019, the cash and stock consideration to be received by Celgene shareholders at closing is valued at $102.43 per Celgene share and one CVR (as described below). When completed, Bristol-Myers Squibb shareholders are expected to own approximately 69 percent of the company, and Celgene shareholders are expected to own approximately 31 percent.
“Together with Celgene, we are creating an innovative biopharma leader, with leading franchises and a deep and broad pipeline that will drive sustainable growth and deliver new options for patients across a range of serious diseases,” said Giovanni Caforio, M.D., Chairman and Chief Executive Officer of Bristol-Myers Squibb. “As a combined entity, we will enhance our leadership positions across our portfolio, including in cancer and immunology and inflammation. We will also benefit from an expanded early- and late-stage pipeline that includes six expected near-term product launches. Together, our pipeline holds significant promise for patients, allowing us to accelerate new options through a broader range of cutting-edge technologies and discovery platforms.”
Dr. Caforio continued, “We are impressed by what Celgene has accomplished for patients, and we look forward to welcoming Celgene employees to Bristol-Myers Squibb. Our new company will continue the strong patient focus that is core to both companies’ missions, creating a shared organization with a goal of discovering, developing and delivering innovative medicines for patients with serious diseases. We are confident we will drive value for shareholders and create opportunities for employees.”
“For more than 30 years, Celgene’s commitment to leading innovation has allowed us to deliver life-changing treatments to patients in areas of high unmet need. Combining with Bristol-Myers Squibb, we are delivering immediate and substantial value to Celgene shareholders and providing them meaningful participation in the long-term growth opportunities created by the combined company,” said Mark Alles, Chairman and Chief Executive Officer of Celgene. “Our employees should be incredibly proud of what we have accomplished together and excited for the opportunities ahead of us as we join with Bristol-Myers Squibb, where we can further advance our mission for patients. We look forward to working with the Bristol-Myers Squibb team as we bring our two companies together.”
Approvals and Timing to Close
The transaction is subject to approval by Bristol-Myers Squibb and Celgene shareholders and the satisfaction of customary closing conditions and regulatory approvals. Bristol-Myers Squibb and Celgene expect to complete the transaction in the third quarter of 2019.
With the start of the new year, planning for 2019’s conferences can begin. HollandBIO members can obtain a discount for industry leading biotech conferences. Take for example the € 300 discount for BIO-Europe Spring on March 25 – 27 in Vienna. In addition, you can save an extra € 200 through an early bird discount when you book before January 21. See the event pagefor all information on BIO-Europe Spring 2019.
HollandBIO members are also eligible for a $ 300 discount for the world’s most influential biotech conference: BIO International Convention, June 3-6 in Philadelphia. Lock in your discount before Friday January 18th by filling out this form. Registration for BIO 2019 is not required at this point. By completing the form, a discount code will be sent to you prior to the registration opening late January 2019. See the event page for all information on BIO 2019.
If you have any questions regarding BIO-Europe Spring of BIO International Convention, please do not hesitate to contact HollandBIO.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-01-03 10:37:002019-01-08 22:17:41Lock in your discounted rate for leading life sciences conferences← #nieuws
DegenRx, a biopharmaceutical company developing novel therapeutic solutions to treat neurodegenerative diseases, has secured financing for their antibody-based gene therapy program for the treatment of Alzheimer’s disease (AD).
Participating in this Series A financing are Swanbridge Capital and BOM Brabant Ventures, together with several private investors seasoned in the pharma and biotech industry. The company will be managed by Guus Scheefhals as Chief Executive Officer and Hans Preusting as Chief Development Officer. DegenRx aims to develop vector-based gene therapies for degenerative brain disorders, such as Alzheimer’s and Parkinson’s disease. Gene therapy aims to provide a potentially lifelong effect after a single administration. DegenRX will develop its proprietary, highly specific antibody, targeting and neutralizing toxic Alzheimer proteins in the brain that would thus slow down or ideally stop disease progression. The current funding will be used to demonstrate the feasibility of this approach in a pre-clinical model.
Guus Scheefhals, CEO of DegenRx, commented: “We are very pleased that the investors are prepared to take on this early opportunity. Degenerative diseases are having an increasing impact on society and most still lack disease-modifying treatments. This investment enables us to show that our innovative approach can work. By making the active compound in the brain, we avoid the need to pass the blood-brain barrier which is typically a significant hurdle for most drugs and certainly for the larger ones, such as antibodies. We are very excited about the potential of our program and hope it will ultimately contribute to the urgently needed benefit for AD patients.”
Nicky Rijk-Vogels, fund manager at Swanbridge Capital: “In terms of phase and technology, DegenRx fits perfectly with the mission of Swanbridge Capital. Our goal is to ensure that young biotech companies can take the next step by providing them with the necessary venture capital and relevant support. We believe in the potential of gene therapy and are happy to contribute to the broader application that DegenRx has in mind for the treatment of degenerative brain diseases. ”
Mercedes Tuin, Investment Manager Life Sciences & MedTech at BOM Brabant Ventures: “BOM Brabant Ventures invests in Brabant companies that have the potential to become disruptive world players. DegenRx has that potential. With Guus Scheefhals and Hans Preusting as Chief Development Officer on board, we have every confidence that the DegenRx team will succeed in their mission.”
Xenikos has partnered with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to conduct a U.S.-based Phase 3 registration trial to test the efficacy of T-Guard in treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following an allogeneic stem cell transplant. Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute, both part of the National Institues of Health, the BMT CTN includes leading transplant centers in the United States (U.S.) and will provide a total of USD 1.37 million in funding for the T-Guard trial.
“At Xenikos, we see BMT CTN’s access to major transplant centers in the U.S., as well as their established track record for successfully recruiting patients with acute GVHD, and vast knowledge and expertise in this field, as aiding in the development of innovative new immunotherapies for improving patient outcomes” said Dr. Ypke van Oosterhout, CEO of Xenikos. “We are therefore confident that our partnership with BMT CTN will help bring T-Guard to transplant patients as quickly as possible.”
“Our network often conducts large, multi-institutional clinical trials in order to test new treatments designed to improve outcomes associated with hematopoietic stem cell transplantation or HSCT,” said Dr. Mehdi Hamadani of the BMT CTN. “Acute GVHD is a life-threatening complication of HSCT and better treatments are needed for our patients. The preliminary data obtained with T-Guard is quite promising, and we believe that the Phase 3 trial is the next logical step.”
The U.S.-based Phase 3 trial will be a multi-center study involving patients who have received an allogeneic stem cell transplant for a myeloid or lymphoid malignancy and subsequently developed SR-aGVHD. Importantly, the Phase 3 trial design is based on input received from the U.S. Food and Drug Administration at the End-of-Phase 2 meeting. Xenikos plans to file an Investigational New Drug application in 2019.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-12-20 11:02:532019-01-08 11:11:39Xenikos will conduct Phase 3 trial to test T-Guard(R) in acute graft-versus-host disease
Groene revolutie maakt 40% hogere oogst mogelijk
Hollandbio, Voedsel en materialenWe staan aan de start van een nieuwe groene revolutie, dankzij een groep Amerikaanse wetenschappers. Door aanpassingen in het DNA van de plant lukte het hen om de groei van planten met 40% te laten toenemen. Dat is een belangrijke doorbraak voor de mondiale voedselproductie. De vraag naar voedsel zal de komende decennia namelijk fors toenemen door een groeiende wereldbevolking, terwijl het areaal landbouwgrond door de klimaatverandering juist afneemt.
Planten zijn in staat om via een ingenieus biologisch proces, de fotosynthese, uit zonlicht en koolstofdioxide (CO2) hun eigen voedingsstoffen (suikers) te maken. Tijdens de fotosynthese komen er naast de nuttige producten zoals suikers en zuurstof (O2), ook voor de plant schadelijke afvalstoffen vrij. Het opruimen van deze schadelijke producten kost de plant veel energie, die ze niet voor haar groei in kan zetten. Door met behulp van genetische modificatie een veel efficiëntere route voor de afbraak van deze afvalstoffen te maken, nam de groei van de plant tot wel 40% toe. De volgende stap is om die groeipotentie gericht te bevorderen in de ‘nuttige’ delen van de plant, bijvoorbeeld de vruchten, zaden, bladeren of stengels. Het bereiken van dit resultaat zal nog wel een aantal jaren duren, net als de toelating van de planten tot de markt. In Europa geldt namelijk een zeer streng, langdurig en onzeker traject voor genetisch gemodificeerde gewassen. HollandBIO pleit al jaren voor aanpassing van deze wetgeving. Deze doorbraak bevestigt maar weer eens hoe belangrijk de modernisering van het biotech beleid is, willen de EU en Nederland straks de vruchten van deze mooie vindingen kunnen plukken.
Bron: Science
Galapagos initiates NOVESA Phase 2a trial in patients with systemic sclerosis
GezondheidGalapagos expands its clinical study program with GLPG1690 in systemic sclerosis, following the recent start of the ISABELA Phase 3 program with ‘1690 in IPF.
NOVESA is a double-blind, placebo-controlled Phase 2a trial evaluating the efficacy, safety and PK/PD of ‘1690 in patients with systemic sclerosis (SSc, or scleroderma). NOVESA is planned to recruit 30 patients with diffuse cutaneous SSc, an autoimmune disease involving multiorgan fibrosis, which has one of the highest mortality rates among rheumatic diseases1 . One of the most visible manifestions is hardening of the skin. In diffuse cutaneous SSc, skin thickening affects several body areas, and patients have a higher risk of developing fibrosis of various internal organs, such as the lung. Currently, there are no approved drugs for this disease. SSc affects approximately 90,000 patients in the US and Europe, with a predominance of female patients (75%).
The primary endpoint of NOVESA is the modified Rodnan skin score (mRSS) at 24 weeks. mRRS measures the skin thickness as a surrogate measure of disease severity and mortality, with an increase in thickness associated with involvement of internal organs and increased mortality2 . Secondary objectives and exploratory endpoints include FVC3 , HRCT4 , quality of life as measured by QoL-Q (SHAQ)5 , and CRISS6 , a SSc disease composite score.
“In addition to our Phase 3 program in IPF7 , we are excited to broaden our development program with ‘1690 to a second indication,” said Dr. Walid Abi-Saab, Chief Medical Officer at Galapagos. “Moreover, SSc is particularly interesting, as this disease straddles our expertise in autoimmune diseases as well as in fibrosis. Thanks to the broad MoA of ‘1690, which is both anti-inflammatory and anti-fibrotic, this compound has the potential to address the important unmet medical need in SSc.”
Source: Galapagos
Fibrocor and Galapagos sign partnership in fibrosis
Gezondheid, InnovatieklimaatFibrocor Therapeutics, a privately held Canadian company, and Galapagos announced a global partnership focused on a novel target for idiopathic pulmonary fibrosis (IPF) and other indications.
Fibrocor specializes in the development of tissue-specific therapeutics to treat the underlying cause of fibrotic diseases of the kidney and other organs. The collaboration announced today concerns a small molecule inhibitor program, currently in the lead optimization stage of development for the treatment of fibrotic diseases of the lung and other organs, the target of which is undisclosed. In exchange for global commercialization rights to Galapagos, Fibrocor will receive an upfront payment, and potentially is eligible for further milestone and royalty payments. Galapagos will be responsible for all further development of the program.
“This collaboration validates the fibrosis drug development expertise of Fibrocor,” says Mark A. Steedman, President and CEO of Fibrocor. “I take my hat off to Dr. Richard Gilbert and the scientific team, including Evotec GmbH, our CRO1 partner, for establishing a compelling data package that ultimately attracted Galapagos, a world-renowned biotech company with a key franchise in fibrosis. We feel this is the beginning of a great relationship and look forward to working with Galapagos to the benefit of fibrosis sufferers everywhere.”
“The collaboration with Fibrocor announced today is an excellent strategic fit for Galapagos, as we continue to expand our franchise in IPF, and more broadly, in fibrosis,” says Dr. Piet Wigerinck, Chief Scientific Officer at Galapagos. “We are enthusiastic about the drug discovery approach at Fibrocor, and look forward to collaborating with the team to address the large unmet medical need in fibrosis.”
Source: Galapagos
Vacature: Management Assistent
Hollandbio32-40 uur, Den Haag
HollandBIO, de belangenvereniging voor biotech bedrijven in Nederland, heeft een duidelijke stip aan de horizon: een samenleving waarin biotech innovaties maximaal bijdragen aan onze gezondheid, een duurzame samenleving en een bloeiende economie.
HollandBIO draagt bij aan het succes van de Nederlandse life sciences sector door bedrijven te vertegenwoordigen en te ondersteunen. We zijn een snelgroeiende vereniging. Een ambitieus en enthousiast team (9 FTE) is verantwoordelijk voor de activiteiten van HollandBIO.
De functie
HollandBIO is op zoek naar een multi-tasker, die zorgt dat alles op rolletjes loopt. Je bent het eerste aanspreekpunt voor externen en de stabiele factor voor het team. Je bent de sparring-partner van de Managing Director en ondersteunt het hele team. Je rapporteert aan de Managing Director.
Secretariële ondersteuning
Administratie
Evenementen
Office management
Jouw profiel
Jij ziet het werk liggen en hebt de dingen al gedaan voordat ze worden gevraagd. Je kunt snel schakelen tussen uiteenlopende activiteiten, hebt een gezonde dosis lef en weet prioriteiten te stellen. Je maakt er een persoonlijke missie van om de perfecte locatie te vinden voor onze events en bijeenkomsten. Je gaat zelfstandig te werk en durft initiatief te tonen. Kortom: we zoeken iemand die alles kan en ook nog eens stressbestendig en gezellig is!
Ons aanbod
Geïnteresseerd?
Stuur voor 15 januari 2019 je motivatie en CV t.a.v. Annemiek Verkamman (Managing Director) naar info@hollandbio.nl. De eerste gesprekken zijn op dinsdag 22 januari en woensdag 23 januari. Voor meer informatie over de functie neem je contact op met Nicole Schönherr (Management Assistent), via telefoonnummer 070-8331333.
De volledige vacture is hier te downloaden
Amicus Therapeutics Announces Phase 1/2 Study of Gene Therapy for CLN3 Batten Disease
GezondheidAmicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, has initiated a Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9 AAV9-CLN3 (AAV9-CLN3) gene therapy in children with CLN3 Batten disease. Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. The initial patient completed a one-month observation period following dosing with no serious adverse events reported to date.
This first in human study of an investigational gene therapy in CLN3 Batten disease, a life-threatening genetic neurologic disorder that typically begins in early childhood and results in premature death, is currently being conducted at Nationwide Children’s Hospital (Columbus, Ohio). CLN3 is the most prevalent of the Batten’s disorders affecting an estimated 5,000+ patients.
“We are pleased to announce that the first child has been dosed in the Phase 1/2 study for CLN3 Batten disease,” said Jay Barth, MD, Chief Medical Officer at Amicus Therapeutics, Inc.“With this pioneering study in CLN3 Batten disease, a severe and devastating neurodegenerative disorder with no approved treatments, we hope that a single intrathecal administration of our gene therapy has the potential to treat the underlying cause of disease and provide a durable and meaningful treatment benefit. This initial participant in our CLN3 Batten disease study, together with the participants in our ongoing clinical study in CLN6 Batten disease, further supports the encouraging safety profile for the intrathecal AAV delivery platform. Advancing this program is another important step forward to potentially improve the lives of thousands of children living with Batten disease and other neurologic lysosomal storage disorders.”
Emily C. De Los Reyes, MD, Principal Investigator at Nationwide Children’s stated, “The initiation of this clinical study in CLN3 Batten disease is an important initial milestone. The proof-of-concept demonstrated in preclinical studies and validation of the intrathecal AAV platform across multiple indications here at Nationwide Children’s Hospital provides promise for the delivery approach in this study. We look forward to enrolling additional participants in the study and comparing the findings to the existing natural history data in CLN3 Batten disease.”
The Phase 1/2 study is enrolling children aged 3 to 10 years with a confirmed diagnosis of CLN3 Batten disease in two sequential intrathecal dose groups: a one-time low-dose of AAV9-CLN3 (Group 1) and a one-time high-dose of AAV9-CLN3 (Group 2) after evaluation of Group 1 participants. Both groups will participate in the current study for a period of three years. Amicus plans to present clinical data from this study, including interim data, at future scientific congresses and other relevant venues. The primary outcome measures are safety and efficacy as determined using the physical disability subscale of the Unified Batten Disease Rating Scale (UBDRS) in CLN3 Batten disease. More information is available at www.clinicaltrials.gov: NCT03770572.
CLN3 Batten disease, the most common form of NCL results from a mutation in the CLN3 gene which primarily affects the nervous system. Children with this condition develop vision impairment, intellectual disability, progressive loss of motor function, speech difficulties, and seizures which worsen over time.
Source: Amicus Therapeutics
ProQR Receives Fast Track Designation from FDA for QR-421a for Usher Syndrome Type 2
Gezondheid, InnovatieklimaatProQR has received a Fast Track designation from the Food and Drug Administration (FDA) for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Fast Track designation is granted by FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.
“We are very pleased with the Fast Track designation the FDA granted us for QR-421a. Patients with Usher syndrome, the leading cause of combined deafness and blindness, currently have no available therapies for their vision loss and this designation emphasizes the high unmet need in this disease,” said Daniel de Boer, Chief Executive Officer of ProQR. “We are also looking forward to begin enrollment in the Phase 1/2 STELLAR clinical trial in the coming months with preliminary data expected in mid-2019.”
Source: ProQR
Bristol-Myers Squibb acquires Celgene
InnovatieklimaatBristol-Myers Squibb and Celgene have entered into a definitive merger agreement under which Bristol-Myers Squibb will acquire Celgene in a cash and stock transaction with an equity value of approximately $74 billion.
Under the terms of the agreement, Celgene shareholders will receive 1.0 Bristol-Myers Squibb share and $50.00 in cash for each share of Celgene. Celgene shareholders will also receive one tradeable Contingent Value Right (CVR) for each share of Celgene, which will entitle the holder to receive a payment for the achievement of future regulatory milestones. The Boards of Directors of both companies have approved the combination.
The transaction will create a leading focused specialty biopharma company well positioned to address the needs of patients with cancer, inflammatory and immunologic disease and cardiovascular disease through high-value innovative medicines and leading scientific capabilities. With complementary areas of focus, the combined company will operate with global reach and scale, maintaining the speed and agility that is core to each company’s strategic approach.
Based on the closing price of Bristol-Myers Squibb stock of $52.43 on January 2, 2019, the cash and stock consideration to be received by Celgene shareholders at closing is valued at $102.43 per Celgene share and one CVR (as described below). When completed, Bristol-Myers Squibb shareholders are expected to own approximately 69 percent of the company, and Celgene shareholders are expected to own approximately 31 percent.
“Together with Celgene, we are creating an innovative biopharma leader, with leading franchises and a deep and broad pipeline that will drive sustainable growth and deliver new options for patients across a range of serious diseases,” said Giovanni Caforio, M.D., Chairman and Chief Executive Officer of Bristol-Myers Squibb. “As a combined entity, we will enhance our leadership positions across our portfolio, including in cancer and immunology and inflammation. We will also benefit from an expanded early- and late-stage pipeline that includes six expected near-term product launches. Together, our pipeline holds significant promise for patients, allowing us to accelerate new options through a broader range of cutting-edge technologies and discovery platforms.”
Dr. Caforio continued, “We are impressed by what Celgene has accomplished for patients, and we look forward to welcoming Celgene employees to Bristol-Myers Squibb. Our new company will continue the strong patient focus that is core to both companies’ missions, creating a shared organization with a goal of discovering, developing and delivering innovative medicines for patients with serious diseases. We are confident we will drive value for shareholders and create opportunities for employees.”
“For more than 30 years, Celgene’s commitment to leading innovation has allowed us to deliver life-changing treatments to patients in areas of high unmet need. Combining with Bristol-Myers Squibb, we are delivering immediate and substantial value to Celgene shareholders and providing them meaningful participation in the long-term growth opportunities created by the combined company,” said Mark Alles, Chairman and Chief Executive Officer of Celgene. “Our employees should be incredibly proud of what we have accomplished together and excited for the opportunities ahead of us as we join with Bristol-Myers Squibb, where we can further advance our mission for patients. We look forward to working with the Bristol-Myers Squibb team as we bring our two companies together.”
Approvals and Timing to Close
The transaction is subject to approval by Bristol-Myers Squibb and Celgene shareholders and the satisfaction of customary closing conditions and regulatory approvals. Bristol-Myers Squibb and Celgene expect to complete the transaction in the third quarter of 2019.
Source: Celgene
Lock in your discounted rate for leading life sciences conferences
HollandbioWith the start of the new year, planning for 2019’s conferences can begin. HollandBIO members can obtain a discount for industry leading biotech conferences. Take for example the € 300 discount for BIO-Europe Spring on March 25 – 27 in Vienna. In addition, you can save an extra € 200 through an early bird discount when you book before January 21. See the event page for all information on BIO-Europe Spring 2019.
HollandBIO members are also eligible for a $ 300 discount for the world’s most influential biotech conference: BIO International Convention, June 3-6 in Philadelphia. Lock in your discount before Friday January 18th by filling out this form. Registration for BIO 2019 is not required at this point. By completing the form, a discount code will be sent to you prior to the registration opening late January 2019. See the event page for all information on BIO 2019.
If you have any questions regarding BIO-Europe Spring of BIO International Convention, please do not hesitate to contact HollandBIO.
DegenRx secures funding for Alzheimer’s disease gene therapy
InnovatieklimaatDegenRx, a biopharmaceutical company developing novel therapeutic solutions to treat neurodegenerative diseases, has secured financing for their antibody-based gene therapy program for the treatment of Alzheimer’s disease (AD).
Participating in this Series A financing are Swanbridge Capital and BOM Brabant Ventures, together with several private investors seasoned in the pharma and biotech industry. The company will be managed by Guus Scheefhals as Chief Executive Officer and Hans Preusting as Chief Development Officer. DegenRx aims to develop vector-based gene therapies for degenerative brain disorders, such as Alzheimer’s and Parkinson’s disease. Gene therapy aims to provide a potentially lifelong effect after a single administration. DegenRX will develop its proprietary, highly specific antibody, targeting and neutralizing toxic Alzheimer proteins in the brain that would thus slow down or ideally stop disease progression. The current funding will be used to demonstrate the feasibility of this approach in a pre-clinical model.
Guus Scheefhals, CEO of DegenRx, commented: “We are very pleased that the investors are prepared to take on this early opportunity. Degenerative diseases are having an increasing impact on society and most still lack disease-modifying treatments. This investment enables us to show that our innovative approach can work. By making the active compound in the brain, we avoid the need to pass the blood-brain barrier which is typically a significant hurdle for most drugs and certainly for the larger ones, such as antibodies. We are very excited about the potential of our program and hope it will ultimately contribute to the urgently needed benefit for AD patients.”
Nicky Rijk-Vogels, fund manager at Swanbridge Capital: “In terms of phase and technology, DegenRx fits perfectly with the mission of Swanbridge Capital. Our goal is to ensure that young biotech companies can take the next step by providing them with the necessary venture capital and relevant support. We believe in the potential of gene therapy and are happy to contribute to the broader application that DegenRx has in mind for the treatment of degenerative brain diseases. ”
Mercedes Tuin, Investment Manager Life Sciences & MedTech at BOM Brabant Ventures: “BOM Brabant Ventures invests in Brabant companies that have the potential to become disruptive world players. DegenRx has that potential. With Guus Scheefhals and Hans Preusting as Chief Development Officer on board, we have every confidence that the DegenRx team will succeed in their mission.”
Source: DegenRx press release
Xenikos will conduct Phase 3 trial to test T-Guard(R) in acute graft-versus-host disease
Gezondheid, InnovatieklimaatXenikos has partnered with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to conduct a U.S.-based Phase 3 registration trial to test the efficacy of T-Guard in treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following an allogeneic stem cell transplant. Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute, both part of the National Institues of Health, the BMT CTN includes leading transplant centers in the United States (U.S.) and will provide a total of USD 1.37 million in funding for the T-Guard trial.
“At Xenikos, we see BMT CTN’s access to major transplant centers in the U.S., as well as their established track record for successfully recruiting patients with acute GVHD, and vast knowledge and expertise in this field, as aiding in the development of innovative new immunotherapies for improving patient outcomes” said Dr. Ypke van Oosterhout, CEO of Xenikos. “We are therefore confident that our partnership with BMT CTN will help bring T-Guard to transplant patients as quickly as possible.”
“Our network often conducts large, multi-institutional clinical trials in order to test new treatments designed to improve outcomes associated with hematopoietic stem cell transplantation or HSCT,” said Dr. Mehdi Hamadani of the BMT CTN. “Acute GVHD is a life-threatening complication of HSCT and better treatments are needed for our patients. The preliminary data obtained with T-Guard is quite promising, and we believe that the Phase 3 trial is the next logical step.”
The U.S.-based Phase 3 trial will be a multi-center study involving patients who have received an allogeneic stem cell transplant for a myeloid or lymphoid malignancy and subsequently developed SR-aGVHD. Importantly, the Phase 3 trial design is based on input received from the U.S. Food and Drug Administration at the End-of-Phase 2 meeting. Xenikos plans to file an Investigational New Drug application in 2019.