ARM Releases Agenda for 2020 Cell & Gene Meeting on the Mediterranean
February 13, 2020 – (ARM) – The event, to be held April 15-17, 2020, in Barcelona, Spain, is modeled after ARM’s highly successful Cell & Gene Meeting on the Mesa and is expected to attract more than 500 attendees. The agenda includes a keynote address from EMA Executive Director Guido Rasi, as well as panels on key topics including hospital exemption, ATMP financings, and cell and gene therapies for chronic conditions.
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Adverum Biotechnologies Announces Pricing of Public Offering of Common Stock
February 12, 2020 – (Adverum Biotechnologies) – Before deducting the underwriting discounts and commissions and estimated offering expenses, Adverum expects to receive total gross proceeds of approximately $130.6M.
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AIFA Authorizes New Areas of Bresso Facility for GMP Manufacturing of Viral Vectors
February 12, 2020 – (MolMed) – MolMed announced that AIFA authorized new areas of the Bresso site for the GMP manufacturing of viral vectors to be used in clinical trials based on advanced therapies.
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AVROBIO Announces Pricing of Underwritten Public Offering of $100M of Common Stock
February 12, 2020 – (AVROBIO) –AVROBIO intends to use the net proceeds from the offering to fund its current programs in Fabry disease, cystinosis, Gaucher disease, and Pompe disease, as well as manufacturing, process development activities, and R&D.
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PolarityTE Announces Pricing of Public Offering of Common Stock and Warrants
February 12, 2020 – (PolarityTE) – PolarityTE intends to use the net proceeds from the $25M offering for commercialization of SkinTE; research, development, and manufacturing of its products and product candidates; efforts toward commercialization and required registration or approval of its products and product candidates with applicable regulatory authorities; and for other general corporate purposes.
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Genprex and University of Pittsburgh Sign Exclusive License Agreement for Potentially Curative Gene Therapy Candidate for Diabetes
February 11, 2020 – (Genprex) – In studies of diabetic mice, the gene therapy approach restored normal blood glucose levels for an extended period of time, typically around four months.
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Orgenesis Completes Sale of Masthercell Subsidiary
February 11, 2020 – (Orgenesis) –Orgenesis announced the completion of the sale of its Masthercell Global subsidiary, a contract development manufacturing organization, to Catalent Pharma Solutions. Upon closing, Orgenesis received net proceeds of approximately $127M from the transaction.
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WindMIL Therapeutics and Providence Cancer Institute Announce Collaboration to Collect Bone Marrow from Patients with Breast Cancer to Develop Marrow-Infiltrating Lymphocytes
February 11, 2020 – (WindMIL) – WindMIL Therapeutics and Providence Cancer Institute announced that the first patient has been identified in an investigator-sponsored study for the collection of bone marrow from patients with breast cancer. The study will evaluate generating marrow-infiltrating lymphocytes for patients through WindMIL’s proprietary cellular activation and expansion process.
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Adaptimmune Therapeutics Announces Full Exercise and Closing of Underwriters’ Option to Purchase Additional American Depositary Shares
February 7, 2020 – (Adaptimmune) – After giving effect to the option exercise, Adaptimmune generated net proceeds of approximately $89.8M. Adaptimmune intends to use the net proceeds from this offering to advance the development of its immunotherapies into and through clinical trials as well as for other general corporate purposes.
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CMRI Announces Major International Collaboration in Gene Therapy
February 5, 2020 – (CMRI) – GE Healthcare Life Sciences has teamed up with Children’s Medical Research Institute in Westmead, Australia to improve the efficiency and scalability of manufacturing of gene therapy tools to fast-track their accessibility on a global scale.
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Sernova Achieves Positive Efficacy Endpoint in Phase 1/2 Clinical Trial for Type 1 Diabetes
February 13, 2020 – (Sernova) – An explanted sentinel pouch showed abundant viable, organized islet cells intimately associated with blood vessels within a collagen matrix after 90 days of transplantation. These surviving islet cells strongly expressed insulin. Sernova announced positive DSMB recommendation for the continuation of their Phase 1/2 clinical trial earlier this week.
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Abeona Therapeutics Announces Positive Interim Data from MPS III Gene Therapy Programs Presented at WORLDSymposium
February 12, 2020 – (Abeona Therapeutics) – Results from the Transpher A study demonstrated that MPS IIIA patients younger than 30 months treated with ABO-102 in dose cohort 3 continue to show neurocognitive development 18 months to two years after treatment. Results from the Transpher B study showed that ABO-101 also improved multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB.
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AlloVir Receives the EMA's PRIME Designation for Viralym-M, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T-Cell Therapy
February 12, 2020 – (Elevate Bio) – AlloVir announced that the EMA has granted PRIority MEdicines (PRIME) designation to Viralym-M for the treatment of serious infections with BK virus, cytomegalovirus, human herpes virus-6, Epstein Barr virus, and/or adenovirus in allogeneic hematopoietic stem cell transplantation recipients.
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Freeline Therapeutics Announces Data on Its Gene Therapy Programs for Fabry Disease and Gaucher Disease
February 12, 2020 – (Freeline Therapeutics) – The infusion was well tolerated. A 3 to 4-fold increase in plasma αGLA activity was achieved by week 4 and sustained through the data cutoff at week 20.
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Prevail Therapeutics’ PR001 Receives Orphan Drug Designation and Rare Pediatric Disease Designation from FDA
February 12, 2020 – (Prevail Therapeutics) – Prevail Therapeutics announced that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation for the company’s investigational gene therapy, PR001, for the treatment of patients with Gaucher disease. Prevail is proceeding with its Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020.
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Marker Therapeutics Announces Update to Clinical Program in AML
February 11, 2020 – (Marker Therapeutics) – Marker Therapeutics announced that the US FDA has lifted the clinical hold on Marker’s planned trial investigating safety and efficacy of its novel MultiTAA T cell therapy in patients with post-transplant acute myeloid leukemia.
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AVROBIO Presents Positive Initial Data for Its Investigational Cystinosis Program and Positive Data Out to 32 Months for Its Ongoing Investigational Fabry Program
February 10, 2020 – (AVROBIO) – Data from the first patient treated in the cystinosis trial shows that the patient’s average granulocyte cystine level decreased from 7.8 nmol half cystine/mg protein to 1.5 at three months post-gene therapy. At nine, 12 and 18 months after dosing, data from three Fabry disease patients indicate sustained increased leukocyte and plasma enzyme activity, suggesting that they are now producing an endogenous supply of functional AGA enzyme.
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LogicBio Therapeutics Provides Update on FDA Review of IND Application for LB-001 for Methylmalonic Acidemia
February 10, 2020 – (LogicBio) – LogicBio Therapeutics announced that the US FDA has placed a clinical hold on the IND submission for LB-001 for the treatment of methylmalonic acidemia pending the resolution of certain clinical and nonclinical questions. The Company submitted the IND in January 2020 to support the initiation of a Phase 1/2 clinical trial in patients with MMA.
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US FDA Grants Priority Review for Kite’s KTE-X19 BLA in Relapsed or Refractory Mantle Cell Lymphoma
February 10, 2020 – (Gilead) – The Prescription Drug User Fee Act, or target action date, is August 10, 2020. EMA recently validated the MAA for KTE-X19 in the European Union.
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Adverum Biotechnologies Reports New Interim Data from Cohorts 1 and 2 of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
February 8, 2020 – (Adverum Biotechnologies) – All six patients in Cohort 1 are rescue-injection-free, with three patients at 52 weeks; 4 of 6 patients in cohort 2 (lower dose cohort) are rescue-injection-free at 24 weeks.
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Gene Therapy/Gene Editing Combo Could Offer Hope for Some Genetic Disorders
February 12, 2020 – (Children's National Hospital) – Researchers created a viral vector that carried an enzyme necessary to create a targeted break in DNA; a second vector then delivered a copy of the correct OTC gene sequence. The new gene successfully integrated in cells and expanded in patches in their livers as the models grew, producing successively more of the necessary detoxifying enzyme.
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New Gene Therapy Method Improves Vision in Mice With Congenital Blindness
February 7, 2020 – (Tohoku University) – The single AAV gene therapy platform combines CRISPR-Cas9 technology with micro-homology-mediated end joining. In blind mice, this approach rescued approximately 10% of photoreceptors, resulting in improved light sensitivity and an increase in visual activity.
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CRISPR-Edited Immune Cells Can Survive and Thrive After Infusion Into Cancer Patients
February 6, 2020 – (University of Pennsylvania) – Researchers showed cells removed from patients and brought back into the lab setting were able to kill cancer months after their original manufacturing and infusion. Further analysis of these cells confirmed they were successfully edited in three specific ways, marking the first-ever sanctioned investigational use of multiple edits to the human genome.
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German Courts Order Health Insurers to Reimburse Zolgensma
February 12, 2020 – (Politico Pro) – Two German courts have ruled that health insurers must reimburse the cost of gene therapy Zolgensma for two children living with spinal muscular atrophy. Explaining its decision, the Berlin Social Court said that the child was about to reach two years of age, which is the cut-off age for Zolgensma approval in the US.
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National Institutes of Health Would See 7% Cut in 2021 Under White House Plan
February 10, 2020 – (Science Magazine) – President Trump’s 2021 budget proposal would slash the NIH budget by $3B to $38.7B, a 7% cut, while maintaining funding for a few priorities. $30M would go toward speeding the manufacture of vectors, such as modified viruses, for delivering gene therapy and gene-editing tools into the body.
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A Lottery Like No Other Offers Up a Cutting-Edge Medicine — With Lives on the Line
February 7, 2020 – (STAT News) – Novartis designed a lottery program for their gene therapy Zolgensma for the treatment of spinal muscular atrophy to give families a chance to get it through a novel form of compassionate use – a way to get medications that have not been approved – while they wait for the therapy to be approved in countries outside the US.
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Director / Vice President, Finance and Operations
Alliance for Regenerative Medicine – Washington, DC
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Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
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