Stem Cell Clinic

Laura Beil’s New Podcast Bad Batch Sheds Light on the World of Unregulated Stem Cell Treatment – D Magazine

Dallas resident Laura Beils podcast Bad Batch is all about giving patients the information they need about an increasingly popular medical treatment that shows both promise and a worrying lack of oversight. The stem cell industry is sometimes hailed as being able to cure everything from male pattern baldness to knee ligament tears, but the lack of oversight and proper research has had disastrous results for patients all over the country.

Beils new project builds on her first podcast, Dr. Death, which covered a Dallas neurosurgeon who paralyzed and killed patients in the operating room, and has now become the first doctor to be convicted for aggravated assault because of care provided in the operating room (read Matt Goodmans D Magazine feature, which first brought the case to light, here). Beils latest installment, which began dropping episodes this month, is another story of vulnerable patients facing harm because they dont have all the information they need to make an informed decision about their healthcare.

Beil had been looking to tell the story of stem cells for a while and was waiting for the opportunity share it via podcast. Its a huge industry, and people are paying their life savings for it, she says. Most of the information people are getting is coming from the people trying to sell it. My job is to tell a different story that could help people.

The podcast, published by Wondery, zooms in on a South Texas clinic that treated several patients with a bad batch of stem cells, leaving several people in the hospital, while also analyzing an industry that has been miracle cure for many. The Texas patients were attracted to the treatment by a local newspaper column where the author sang the praises about how the treatment healed her leg.

The stories in the podcast range from rural Wisconsin residents successfully healed by stem cells to a biomedical consultant in Thailand who left a stem cell research company after he decided that they werent operating in a way that would result in safe and effective stem cells.

But because of all the significant success and legitimate research behind stem cells, the narrative behind Bad Batch is unlike true crime podcasts with a clear culprit, Beil says. Alongside the above-board treatment and research, there is a retail industry springing up selling unapproved therapy to people.

Texas business-friendly environment has made it a hotbed of stem cell treatment, but the lack of regulation and oversight can have real consequences. Texas has arguably the laxest stem cell regulations are in Texas, Beil says. Texas has embraced unapproved stem cell treatment.

Biels transition from science and health journalist operating mostly in print to the voice and reporting behind some of the most popular podcasts in the country has been an engaging one, and she is still learning about how to best tell an audio story.

In audio, one thing I have learned, and am still trying to learn, is that you only have one shot to interview people, she says. In print, you can get more detail or clarify, and you can email or call them back to ask details. In audio you cant do that.

Beil hopes that her story balances entertainment with impact and allows listeners to make a more informed decision should they go down the stem cell path. It is a good story to tell, and as a journalist that is my job, but at the same time I want to tell a story that is in the public service, she says.

These remedies that are being sold have not been studied like a drug would, but people doing it make the assumption it is safe and effective. People dont know how many unknowns there are.

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Laura Beil's New Podcast Bad Batch Sheds Light on the World of Unregulated Stem Cell Treatment - D Magazine

Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting – Business…

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, today announced that hemophilia A gene therapy clinical data and hemoglobinopathies ex vivo gene-edited cell therapy data will be featured in poster presentations at the 61st Annual Meeting of the American Society of Hematology (ASH). The ASH abstracts, which were submitted on August 3, 2019, were released online this morning. The conference will take place in Orlando, FL, from December 7-10, 2019.

Gene Therapy

The SB-525 poster will show updated Alta study data including durability of Factor VIII (FVIII) levels, bleeding rate, factor usage, and safety, for all five patients in the high dose cohort of 3e13 vg/kg, with approximately 4 months to 11 months of follow-up after treatment with SB-525.

As of the abstract submission date, four patients in the 3e13 vg/kg cohort achieved FVIII levels within the normal range with no bleeding events reported up to 24 weeks post-administration. These patients did not require FVIII replacement therapy following the initial prophylactic period of up to approximately 3 weeks post-SB-525 administration. The fifth patient in the 3e13 vg/kg cohort had only recently undergone treatment with SB-525 at the time of the abstract submission. As previously reported, one patient had treatment-related serious adverse events (SAEs) of hypotension and fever, which occurred approximately 6 hours after completion of the vector infusion and resolved with treatment within 24 hours, with no loss of FVIII expression. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.

The rapid kinetics of Factor VIII expression, durability of response, and the relatively low intra-cohort variability in the context of a complete cessation of bleeding events and elimination of exogenous Factor VIII usage continues to suggest SB-525 is a differentiated hemophilia A gene therapy, said Bettina Cockroft, M.D., M.B.A., Chief Medical Officer of Sangamo, commenting on the published abstract. We are pleased with the progress of the program toward a registrational Phase 3 study led by Pfizer, who announced it has enrolled its first patient in the 6-month Phase 3 lead-in study. We have recently completed the manufacturing technology transfer to Pfizer and initiated the transfer of the IND.

Ex Vivo Gene-Edited Cell Therapy

The ST-400 beta thalassemia poster will show preliminary results from the first three patients enrolled in the Phase 1/2 THALES study. In this study, hematopoietic stem progenitor cells (HSPCs) are apheresed from the patient, edited to knock out the erythroid specific enhancer of the BCL11A gene, and cryopreserved prior to infusion back into the patient following myeloablative conditioning with busulfan. The first three patients all have severe beta thalassemia genotypes: 0/0, homozygous for the severe + IVS-I-5 (G>C) mutation, and 0/+ genotype including the severe IVS-II-654 (C>T) mutation, respectively.

As of the abstract submission date, Patient 1 and Patient 2 had experienced prompt hematopoietic reconstitution. Patient 1 had increasing fetal hemoglobin (HbF) fraction that contributed to a stable total hemoglobin. After being free from packed red blood cell (PRBC) transfusions for 6 weeks, the patient subsequently required intermittent transfusions. Patient 2 had rising HbF levels observed through 90 days post-infusion. For both patients, as of the most recent follow-up reported in the abstract, on-target insertions and deletions (indels) were present in circulating white blood cells. Patient 3 had just completed ST-400 manufacturing at the time of abstract submission. As previously disclosed, Patient 1 experienced an SAE of hypersensitivity during ST-400 infusion considered by the investigator to be related to the product cryoprotectant, DSMO, and which resolved by the end of the infusion. No other SAEs related to ST-400 have been reported and all other AEs have been consistent with myeloablation. No clonal hematopoiesis has been observed. Longer follow-up will be required to assess the clinical significance of these early results. ST-400 is being developed as part of a global collaboration between Sangamo and Sanofi, along with support through a grant from the California Institute for Regenerative Medicine (CIRM).

The first three patients enrolled in the THALES study all have severe beta thalassemia genotypes that result in almost no endogenous beta globin production. The increases in fetal hemoglobin and presence of on-target indels in circulating blood cells suggests successful editing using zinc finger nucleases. The results are preliminary and will require additional patients and longer-term follow-up to assess their clinical significance, said Adrian Woolfson, BM., B.Ch., Ph.D., Head of Research and Development. It is important to note that myeloablative hematopoietic stem cell transplantation reboots the hematopoietic system, and that sufficient time is required for the stem cells to fully repopulate the marrow and for new blood cells to form. In other myeloablative conditioning studies in a similar patient population, full manifestation of the effects of gene modification in the red blood cell compartment has taken as long as 12 months or more to become evident.

Sanofis in vitro sickle cell disease poster details a similar approach to ST-400, using mobilized HSPCs from normal donors and SCD patients and utilizing the same zinc finger nuclease for gene editing, delivered as transient non-viral RNA, and designed to disrupt the erythroid specific enhancer of the BCL11A gene, which represses the expression of the gamma globin genes, thereby switching off HbF synthesis. Results from ex vivo studies demonstrated enriched biallelic editing, increased HbF, and reduced sickling in erythroid cells derived from non-treated sickle cell disease patients. Sanofi has initiated a Phase 1/2 trial evaluating BIVV003, an ex vivo gene-edited cell therapy using ZFN gene editing technology to modify autologous hematopoietic stem cells using fetal hemoglobin to produce functional red blood cells with higher BhF content that are resistant to sickling in patients with severe sickle cell disease. Recruitment is ongoing.

About the Alta study

The Phase 1/2 Alta study is an open-label, dose-ranging clinical trial designed to assess the safety and tolerability of SB-525 gene therapy in patients with severe hemophilia A. SB-525 was administered to 11 patients in 4 cohorts of 2 patients each across 4 ascending doses (9e11 vg/kg, 2e12 vg/kg, 1e13vg/kg and 3e13vg/kg) with expansion of the highest dose cohort by 3 additional patients. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency.

About the THALES study

The Phase 1/2 THALES study is a single-arm, multi-site study to assess the safety, tolerability, and efficacy of ST-400 autologous hematopoietic stem cell transplant in 6 patients with transfusion-dependent beta thalassemia (TDT). ST-400 is manufactured by ex vivo gene editing of a patient's own (autologous) hematopoietic stem cells using non-viral delivery of zinc finger nuclease technology. The THALES study inclusion criteria include all patients with TDT (0/0 or non- 0/0) who have received at least 8 packed red blood cell transfusions per year for the two years before enrollment in the study. The FDA has granted Orphan Drug status to ST-400.

About Sangamo Therapeutics

Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic medicines with the potential to transform patients' lives using gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing, and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Forward-Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies, as well as the timing of commencement of clinical programs and the anticipated benefits therefrom. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the outcomes of clinical trials, the uncertain regulatory approval process, uncertainties related to the execution of clinical trials, Sangamo's reliance on partners and other third-parties to meet their clinical and manufacturing obligations, and the ability to maintain strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable product candidates. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission and Sangamo's most recent Quarterly Report on Form 10-Q. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

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Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting - Business...

bluebird bio to Present New Data from Gene and Cell Therapy Programs at 61st American Society of Hematology Annual Meeting and Exposition – Financial…

Updated safety and efficacy results from ongoing Phase 1 CRB-402 study of bb21217 in relapsed/refractory multiple myeloma

Updated results from ongoing Phase 1/2 (HGB-206) study of LentiGlobin gene therapy for patients with sickle cell disease

New data from ongoing Phase 3 studies of LentiGlobin gene therapy for -thalassemia in pediatric, adolescent and adult patients

CAMBRIDGE, Mass. bluebird bio, Inc. (Nasdaq: BLUE) announced today that new and updated data from its investigational gene and cell therapy programs for multiple myeloma, sickle cell disease (SCD) and transfusion-dependent -thalassemia (TDT) will be presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida, December 7 10.

bluebird bio will present updated safety and efficacy data from the ongoing Phase 1 clinical study (CRB-402) of bb21217. bb21217 is an investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy being studied, in partnership with Celgene, in patients with relapsed/refractory multiple myeloma (RRMM).

In addition, data from clinical studies of LentiGlobin gene therapy for -thalassemia, including results up to 61 months from the long-term follow-up study (LTF-303) and updated results from the completed Phase 1/2 Northstar (HGB-204) study, will be presented at ASH. The company will also present new data from the ongoing Phase 3 Northstar-2 (HGB-207) study in pediatric, adolescent and adult patients who do not have a 0/0 genotype and from the ongoing Phase 3 Northstar-3 (HGB-212) study in pediatric, adolescent and adult patients who have 0/0 genotype or an IVS-I-110 mutation at both alleles of the -globin gene.

New data from the companys Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will include additional patients treated in the study and updated data for those previously reported. The company will also present data from exploratory assays designed to assess the relationship between drug product characteristics and red blood cell physiology in patients treated with LentiGlobin for SCD.

Updated Data from Ongoing Phase 1 Clinical Study (CRB-402) of bb21217

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 Anti-BCMA CAR T Cell Therapy Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn. Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

bb21217, an investigational BCMA-targeted CAR T cell therapy being developed in partnership with Celgene, is one of bluebird bios lead oncology programs. bb21217 uses the idecabtagene vicleucel CAR molecule (formerly referred to as bb2121) and is manufactured with a process intended to increase the in vivo persistence of CAR T cells.

This presentation will include updated data from the Phase 1 CRB-402 study, the first-in-human study of bb21217 in patients with RRMM, designed to assess the primary endpoint of safety as well as other pre-defined endpoints including efficacy and pharmacokinetics measurements. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients.

Data in the abstract include results as of the data cutoff date of April 20, 2019 for 22 patients who have received bb21217 at three dose levels (12 at 150 x 106 CAR+ T cells; six at 300 x 106 CAR+ T cells; and four at 450 x 106 CAR+ T cells). These patients had a median of seven prior lines of therapy (min-max: 4 17 lines), 18 patients had a prior autologous stem cell transplant, 19 patients received daratumumab and 13 patients received prior treatment with bortezomib, lenalidomide, carfilzomib, pomalidomide and daratumumab.

As of the data cutoff, the adverse events observed were consistent with known toxicities of CAR T therapies. Thirteen of 22 patients developed cytokine release syndrome (CRS); five Grade 1, seven Grade 2, and one Grade 3 case. All 13 patients responded to supportive care, tocilizumab and/or corticosteroids. Five of 22 patients developed neurotoxicity; one Grade 1, two Grade 2, one Grade 3 (vertigo/dizziness), and one Grade 4 (encephalopathy, previously reported). For the one patient previously reported with Grade 4 neurotoxicity, Grade 3 CRS was also reported, and both have resolved.

Eighteen patients were evaluable for clinical response with > two months of follow-up or progressive disease within two months. Eighty-three percent (n=15/18) of evaluable patients demonstrated clinical response per the International Myeloma Working Group Uniform Response Criteria for multiple myeloma. As of the data cutoff, with the median follow-up after bb21217 infusion of five months (min-max: <1 18 months), nine patients remained in response, including two patients with ongoing response at 15 and 18 months.

Evidence of myeloma in the bone marrow, known as minimal residual disease, was undetectable by next-generation sequencing at a sensitivity level of 10-5 or better in all responders who had evaluable bone marrow samples (n=10) at Month 1. CAR T cell persistence was observed in six of eight patients evaluable at six months and in two of two patients evaluable at 12 months.

This study is ongoing to evaluate the potential safety and efficacy of treatment with bb21217, and updated results, including early clinical and CAR T cell persistence data, will be shared at the ASH conference.

Multiple Myeloma Presentations at ASH

Markers of Initial and Long-Term Responses to Idecabtagene Vicleucel (Ide-Cel; bb2121) in the CRB-401 Study in Relapsed/Refractory Multiple Myeloma Presenting Author: Ethan G. Thompson, Ph.D., Celgene, Seattle, Wash. Date & Time: Poster #4328, Monday, December 9, 2019, 6:00 8:00 p.m. ET

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 anti-BCMA CAR T Cell Therapy Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn. Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

SCD Presentations at ASH

The Relationships Between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy Presenting Author: Melissa Bonner, Ph.D., bluebird bio, Cambridge, Mass. Date & Time: Oral #206, Saturday, December 7, 2019, 2:15 p.m.

Exploring the Drivers of Clinical Benefit in Initial Patients Treated in the HGB-206 Study of LentiGlobin for Sickle Cell Disease (SCD) Gene Therapy Presenting Author: Mark Walters, M.D., Benioff Childrens Hospital, Oakland, Calif. Date & Time: Poster #2061, Saturday, December 7, 2019, 5:30 7:30 p.m.

Resolution of Sickle Cell Disease Manifestations in Patients Treated with LentiGlobin Gene Therapy: Updated Results from the Phase 1/2 HGB-206 Group C Study Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala. Date & Time: Poster #990, Saturday, December 7, 2019, 5:30 7:30 p.m.

TDT Presentations at ASH

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent -Thalassemia Treated at the Bambino Ges Childrens Hospital, Rome, Italy Presenting Author: Pietro Merli, M.D., IRCCS Ospedale Pediatrico Bambino Ges, Rome, Italy Date & Time: Poster #969, Saturday, December 7, 2019, 5:30 7:30 p.m.

Northstar-3: Interim Results from a Phase 3 Study Evaluating LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Either a 0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene Presenting Author: Ashutosh Lal, M.D., UCSF Benioff Childrens Hospital, Oakland, Calif. Date & Time: Oral #815, Monday, December 9, 2019, 5:30 p.m.

Northstar-2: Updated Safety and Efficacy Analysis of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Non-0/0Genotypes Presenting Author: Alexis Thompson, M.D., MPH, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, Ill. Date & Time: Poster #3543, Monday, December 9, 2019, 6:00 8:00 p.m.

Long-Term Clinical Outcomes of LentiGlobin Gene Therapy for Transfusion-Dependent -Thalassemia in the Northstar (HGB-204) Study Presenting Author: Janet Kwiatkowski, M.D., MSCE, Childrens Hospital of Philadelphia, Philadelphia, Pa. Date & Time: Poster #4628, Monday, December 9, 2019, 6:00 8:00 p.m.

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent -Thalassaemia in the United Kingdom: A Mixed Methods Observational Study Presenting Author: Farrukh Shah, MBBS, FRCP, FRCPath, M.D., Whittington Hospital, London, U.K. Date & Time: Poster #3550, Monday, December 9, 2019, 6:00 8:00 p.m.

SCD and TDT Presentation at ASH

Results from the Completed HGB-205 Trial of LentiGlobin for -Thalassemia and LentiGlobin for Sickle Cell Disease Gene Therapy Presenting Author: Elisa Magrin, Ph.D., Necker Childrens Hospital, Assistance Publique-Hpitaux de Paris, Paris, France Date & Time: Poster #3358, Sunday, December 8, 2019, 6:00 8:00 p.m.

Abstracts outlining bluebird bios accepted data at ASH will be available on the ASH conference website at 9 a.m. EST today.

About ide-cel and bb21217 for Multiple Myeloma

bluebird bios lead oncology programs, idecabtagene vicleucel (ide-cel, formerly referred to as bb2121) and bb21217, are investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapies being studied in a broad clinical development program for patients with multiple myeloma. ide-cel and bb21217 are being developed in partnership with Celgene.

KarMMa is a registration-enabling, open-label, single-arm, multi-center Phase 2 study evaluating the efficacy and safety of ide-cel in patients with relapsed/refractory multiple myeloma. In November 2018, bluebird bio announced completion of enrollment in the trial. ide-cel was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and Priority Medicines (PRIME) eligibility by the European Medicines Agency in November 2017 based on preliminary clinical data from the Phase 1 CRB-401 study.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with RRMM, designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients. For more information visit: clinicaltrials.gov using identifier NCT03274219.

ide-cel and bb21217 are not approved for any indication in any geography.

About LentiGlobin for Sickle Cell Disease

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/ or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About LentiGlobin for -Thalassemia

The European Commission granted conditional marketing authorization for LentiGlobin for TDT, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or absent hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived-hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to LentiGlobin for TDT were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/ or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Companys views with respect to the potential for LentiGlobin to treat transfusion-dependent -thalassemia and sickle cell disease, the potential for the bb21217 product candidate to treat relapsed/ refractory multiple myeloma. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of our product candidates will not continue or be repeated in our ongoing or planned clinical trials or in the commercial context, risks that the current or planned clinical trials of our product candidates will be insufficient to support future regulatory submissions or to support marketing approval in the US and EU, and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191106005511/en/

Contacts

Investors: Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com or Media: Catherine Falcetti, 339-499-9436 cfalcetti@bluebirdbio.com

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bluebird bio to Present New Data from Gene and Cell Therapy Programs at 61st American Society of Hematology Annual Meeting and Exposition - Financial...

Three Podcasts to Listen to in November – The New Yorker

This fall brings a fresh crop of documentary podcast series, many of them timely but not overfamiliar. Tunnel 29, the new season of BBC Radio 4s Intrigue series, reported and narrated by Helena Merriman, tells the true story of Joachim Rudolph, who, in the early sixties, as a twenty-two-year-old engineering student in communist East Germany, escaped to West Berlinand then dug a tunnel back into East Germany, beneath the Berlin Wall, to help other refugees escape. Theres plenty of spade-and-dirt action, and panting, in this Stasi-haunted dramatization of heroism, as Rudolph and an unlikely team of helpers burrow toward an East German basement. But, unlike last seasons narratively unnerving Intrigue: The Ratline (upsetting Schloss, Nazi horrors, typewriter sounds), Tunnel 29 finds a deft balance of realism and drama in its narration, performance, and sound design. A surprisingly lovely toast-eating scene, featuring pineapple marmalade and freedom, packs an unexpected punch.

Bad Batch, from the reporter Laura Beil, could fit into two genres. The first is medical true crime, as pioneered by Beil in her previous series, Dr. Death, about a horrifically injurious Texas neurosurgeon; the second is the startup cautionary tale, la The Dropout, about Elizabeth Holmes and Theranos. Bad Batch delves into the promising but murky realm of stem-cell medical treatments, beginning with a deep dive into an incident in which treatments using a bad batch of stem cells, originating from a single company, almost killed several people. Beil, who hosts, takes us through backstories involving health-care fraud, a Ponzi scheme, and high-pressure stem-cell educational seminars, which hoodwink vulnerable people into spending thousands on dubious therapies. Four episodes in, Bad Batch has enlightened me more about fraudsters than science; future episodes may go further to sort out stem-cell fact from fiction. Meanwhile, Im pleased that this series, produced, like Dr. Death, by the sensationalism-prone hitmaker Wondery, so far avoids the noir impulses of its predecessor.

Like Nashville Public Radios excellent The Promise, about a public-housing gambit in Nashville, the new season of USA Todays The City, featuring Robin Amer and Anjeanette Damon, tells the story of a fast-changing city via one vivid enclave within it; here, its Reno, Nevada, and its strip clubs. A tech boom in Reno, near Elon Musks Tesla Gigafactory 1, has driven a real-estate frenzy and a gentrification battle, and The City takes us inside itso far, to city-council meetings, private-eye stakeouts, and a newly vulnerable residential hotel. The reporting has a sensitive ear for distinctive characters, like Velma Shoals, a hotel resident determined to save her home, and Kamy Keshmiri, a straight-talking Reno native and former athlete whose family owns the hotel and a strip club. (Its sad that youre bringing private investigators in to look at boobs, he says at one point, about officials hoping to catch illegal activity at the club. Yeahtheres boobs.) The series provides an impressively sweeping but intimate look at an American city; I began it knowing little about Reno and emerged wanting to know everything.

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Three Podcasts to Listen to in November - The New Yorker

Celularity to Present New Data at the ASH Annual Meeting on Novel Allogeneic, Off the Shelf, Placental Derived CAR T and NK Cell Therapy Programs -…

WARREN, N.J.--(BUSINESS WIRE)--

Five abstracts to be presented on the pre-clinical and clinical results, demonstrating the breadth and versatility of the Companys allogeneic, off the shelf, placental derived cell therapy platform in multiple myeloma and lymphoma

Celularity, Inc., a clinical-stage cell therapeutics company developing allogeneic cellular therapies harnessed from human placentas, today announced it will present data supporting its allogeneic, off-the-shelf, placental derived cell therapy programs at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 7-10 at the Orange County Convention Center in Orlando, Florida.

We believe the placenta is natures cell factory, and the data being presented at ASH will illustrate the potential of our investigational placental derived NK- and T cell-based allogeneic cell therapy programs in oncology, said Robert J. Hariri, M.D., Ph.D., Founder, Chairman and CEO at Celularity. We look forward to advancing Celularitys leading-edge technologies that harness the placentas unique immunologic and pro-regenerative biology to produce therapeutic solutions targeting unmet healthcare needs globally.

ASH abstracts are now available at https://www.hematology.org/.

Details for the 2019 ASH poster presentations are as follows:

Title: Development of CD38 CAR Engineered Human Placental Hematopoietic Stem Cell Derived Natural Killer Cells (PNK-CAR38) As Allogeneic Cancer Immunotherapy Date: Saturday, December 7, 2019

Title: Engineering High Affinity and Cleavage Resistant CD16 to Augment ADCC of Placental Hematopoietic Stem Cells-Derived Natural Killer Cells Date: Saturday, December 7, 2019

Title: Preclinical Evaluation of Human Placental-Derived Allogeneic CD19 CAR-T Cells Against B Cell Malignancies Date: Sunday, December 8, 2019

Title: Results of a Phase I Study of PNK-007, Allogeneic, Off the Shelf NK Cell, Post Autologous Transplant in Multiple Myeloma (NCT02955550) Date: Monday, December 9, 2019

Title: Immune Monitoring of CD34+ Placental Cell Derived Natural Killer Cell Therapy (PNK-007) in Phase I Study of Multiple Myeloma Date: Monday, December 9, 2019

About PNK-007 PNK007 is the only allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. When derived from the placenta, these cells offer intrinsic safety and versatility, allowing potential use across a range of organs and tissues. PNK cells are currently being investigated as a treatment for acute myeloid leukemia (AML) and multiple myeloma (MM).

About CYNK-001 CYNK-001, a cryopreserved formulation of PNK-007 cells, is the only cryopreserved, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors.

About Celularity Celularity, headquartered in Warren, New Jersey, is a clinical-stage cell therapeutics company delivering transformative allogeneic cellular therapies, engineered from the postpartum human placenta. Using proprietary technology in combination with its IMPACT platform, Celularity is the only company harnessing the purity and versatility of placental derived cells to develop and manufacture innovative and highly scalable off-the-shelf treatments for patients with cancer, inflammatory and age-related diseases. To learn more, please visit http://www.celularity.com.

Forward-Looking Statements This press release contains forward-looking statements. These forward-looking statements are based on expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. The information contained in this press release is believed to be current as of the date of original issue. Celularity expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191106005565/en/

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Celularity to Present New Data at the ASH Annual Meeting on Novel Allogeneic, Off the Shelf, Placental Derived CAR T and NK Cell Therapy Programs -...

19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results – Cancer Therapy Advisor

According to results of a study published in Blood, children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) who had minimal residual disease (MRD) prior to treatment and received high-dose preconditioning chemotherapy were most likely to respond to a second-generation CD-19 chimeric antigen receptor-T cell (CAR-T) therapy.1

Although it has been estimated that 90% or more of pediatric patients witha diagnosis of ALL will respond to multi-agent chemotherapy, the prognosis forthose with relapsed/refractory disease remains poor. One CD19-directed CAR-Ttherapy, tisagenlecleucel, is approved by the US Food and Drug Administration inpatients up to age 25 years with B-cell precursor ALL who either have refractorydisease or have experienced a second or later relapse.2

This open label, nonrandomized, phase 1 study (Clinical Trial Identifier: NCT01860937), evaluated the toxicity, feasibility, and response of 19-28z CAR-T therapy, a second-generation CD19-directed CAR-T therapy involving T cells expressing a chimeric receptor composed of an anti-CD19 antibody binding site and intracellular domains from the T-cell coactivating receptors, CD28 and the CD3-zeta chain3 in children and young adults up to 25 years of age with very high-risk ALL.1 Inclusion criteria included at least 2 relapses, early bone marrow relapse following complete response (CR), intermediate/late CR with poor response to re-induction therapy, or those with refractory disease, or ineligibility for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or additional chemotherapy.1

The age range of the 25 patients treated with 19-28z CAR-T therapy onstudy was 1 to 22.5 years, with a median age of 13.5 years. Preconditioningchemotherapy involved high-dose cyclophosphamide (15 patients) and low-dosecyclophosphamide (8 patients), with 3 patients in each subgroup also receivingfludarabine.1

Regarding the feasibility of this approach, the prespecified CAR-Tcell dose was achieved for all patients for whom the 19-28z CAR-T therapyprocedure was undertaken.1

With respect to treatment toxicity,approximately one-third of patients experienced a grade 3/4 adverse event,including cytokine release syndrome (CRS) and neurotoxicity in 16% and 28% ofpatients, respectively. With the exception of 1 patient with grade 4 CRS andneurotoxicity who died following refractory Stenotrophomonas septic shock,these adverse events were reversible.1

Of the 24 patients includedin the response analysis, 75% achieved either a CR or a CR with incompletecount recovery (CRi). In the subsets of patients receiving preconditioningchemotherapy with either high- or low-dose cyclophosphamide, the CR/CRi rateswere 94% and 38%, respectively. Furthermore, treatment response was influencedby disease burden as evidenced by the considerably higher CR/CRi rate inpatients with baseline minimal residual disease (ie, less than 5% bone marrowblasts; 93%) compared with morphological evidence of disease at baseline (5% orhigher bone marrow blasts; 50%).1

The CR/CRi rate for thesubset of patients with pretreatment MRD treated with high-dose cytarabine was100%.1

Consolidation allo-HSCT was performed in 83% (15) of the patientsresponding to CAR-T therapy, with a median time from CAR-T infusion toallo-HSCT of 57 days. At a median follow-up of 28.6 months for respondingpatients, over half of these patients (8) were alive and had no evidence ofdisease.1

In their concluding remarks, the study authorscommented that thisanalysis has allowed us to determine the toxicity profile, confirm feasibility,evaluate response of this approach, and provide a direct comparison of the sameCD19-specific CAR T cell product that was previously published[3] inadult patients for the same indication.

The authors went on to highlight the findingof a reversible toxicity profile in the patients within their study as well asthe impact of preconditioning chemotherapy dose intensity and minimal pretreatmentdisease burden on response.

They further noted that within this cohort,the long-term persistence of response is encouraging, and in our primarilytransplant-naive patient population, the ability to proceed to allo-HSCT hasdemonstrated a favorable overall survival, manageable toxicity, and limitedincidence of relapse.

References

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19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results - Cancer Therapy Advisor

MD Anderson on a Roll with Another Pharma Deal, This Time with Artios and ShangPharma – BioSpace

Only a day after The University of Texas MD Anderson Cancer Center announced it has partnered with Takeda Pharmaceutical on immuno-oncology therapies, MD Anderson announced it was involved in an in-licensing deal with Artios Pharma and ShangPharma.

Under the new deal, Artios Pharma licensed rights to research, develop, manufacture and commercialize products globally from a small-molecule ATR inhibitor program that was jointly developed by MD Anderson and ShangPharma. Artios Pharma, based in Cambridge, UK, plans to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) by the second half of 2020.

ATR is a signaling protein in DNA double-strand break repair and replication stress. Inhibiting ATR can kill tumors that have an ATM deficiency. This occurs through a process dubbed synthetic lethality. Many types of cancers have high levels of ATM mutations and protein loss, which makes them particularly promising targets for cancer therapeutics.

We are proud of the entire collaboration team, including ChemPartner, led by Sarah Lively, PhD, vice president of Innovation and New Technologies, for advancing the program from early-stage research to formal drug discovery and development, said Walter Moos, chief executive officer of ShangPharma, which is based in China and San Francisco. We are pleased to transition this important program to the capable development team at Artios, and we hope this ultimately provides an impactful therapy for those afflicted with cancer.

The ATR inhibitor program is the results of a collaboration between MD Andersons Therapeutics Discovery group and ShangPharma. The Therapeutics Discovery team was created within MD Anderson to promote the next generation of cancer drugs.

After clinical studies at MD Anderson, Therapeutics Discovery worked with ShangPharma and its affiliate, ChemPartner, to develop small-molecule inhibitors of the DNA Damage response (DDR) that would be effective across several cancer types.

Targeting DNA damage repair has the potential to provide an important therapeutic option for many patients in need of new treatments, said Philip Jones, vice president of Therapeutics Discovery at MD Anderson. We are pleased Artios will leverage its unique expertise in this field to advance this novel therapy toward the clinic to improve outcomes for cancer patients.

No financial details were disclosed.

Yesterday, MD Anderson and Takeda announced an exclusive license deal and research agreement to develop cord-blood derived chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies. They say these CAR-NK therapies will be armored with IL-15 to treat B-cell and other cancers.

Under the deal, Takeda will access MD Andersons CAR-NK technology platform and pick up the exclusive rights to develop and commercialize up to four programs. Those programs include a CD19-targeted CAR-NK-cell therapy and a B-cell maturation antigen (BCMA)-targeted CAR-NK therapy. They will collaborate on research to advance the programs.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly and with minimal toxicities, said Katy Rezvani, professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. With their expertise in hematologic malignancies and commitment to developing next-generation cell therapies, Takeda is the ideal collaborator to help our team advance CAR NK-cell therapies to patients in need of treatments.

MD Andersons allogeneic CAR NK technology platform collects umbilical cord blood, isolates NK cells for it, and then engineers those NK cells to express CARs against specific cancer targets. They utilize a retroviral vector to deliver genes to the CAR NK cells, which both improves their effectiveness and fine-tunes them for specific cancer cells. The CD19 CAR makes the cells even more specific for B-cell malignancies, and the IL-15 improves the proliferation and survival of the CAR-NK cells in the body.

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MD Anderson on a Roll with Another Pharma Deal, This Time with Artios and ShangPharma - BioSpace

Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting -…

Registrational Trial for Wiskott-Aldrich Syndrome Met Key Primary and Secondary Endpoints at Three Years; Data from Integrated Analysis Reinforce Treatment Benefits of Gene Therapy and Durability of Effect in Additional Patients

Similar Profiles Reported Between Cryopreserved and Fresh Formulations of OTL-101, Further Supporting Upcoming Regulatory Filing and Broad Patient Availability

BOSTON and LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced the upcoming presentation of registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, FL.

Investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at theSan Raffaele-Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, investigators will deliver an oral presentation featuring updated data from the ongoing proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) atSR-Tiget.

This growing body of positive data, from dozens of patients across multiple diseases, provides a solid foundation as we advance each program toward its next phase of development, including upcoming regulatory submissions for ADA-SCID and WAS, saidMark Rothera, president and chief executive officer ofOrchard Therapeutics. We now have two supportive data sets one from our OTL-101 program in ADA-SCID and one from our OTL-200 program in metachromatic leukodystrophy that demonstrate cryopreserved formulations are engrafting as expected, similar to the fresh formulation. This supports our strategy for making these therapies, if approved, broadly available to patients in need throughout the world.

We are extremely pleased with our continued clinical progress, including the duration of benefits seen in our WAS trial, which is the longest published follow-up of hematopoietic stem cell gene therapy durability to date using lentiviral vector transduction, said Bobby Gaspar, M.D., Ph.D., chief scientific officer of OrchardTherapeutics. The totality of these data underscores the broad applicability of our gene therapy platform approach and the opportunity we have to deliver potentially curative treatments for a variety of devastating and rare genetic disorders.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time:Sunday, December 8, 6:00-8:00pm ET

This presentation includes results from an integrated analysis of 17 patients treated with OTL-103 for the treatment of WAS, including the complete data set for the eight patients from the registrational study and nine who received OTL-103 as part of an expanded access program (EAP). Participants have been followed for a median of three years.

In the eight-patient registrational trial, investigators reported that the study achieved its key primary and secondary endpoints at three years, including the elimination of severe bleeding episodes and a significant reduction in the frequency of moderate bleeding episodes. Successful engraftment was observed within three months, leading to an increase in WAS protein expression and a vector copy number that has been maintained for up to eight years. Nine months post-administration, all patients stopped receiving platelet transfusions, and no severe bleeding events were reported. A significant reduction in the rate of severe infections was also observed and all patients were able to stop immunoglobin replacement therapy (IgRT), suggesting a complete reconstitution of immune function with durability of effect of up to eight years of follow-up post-gene therapy.

Similar clinical results were seen in the integrated analysis of 17 patients and overall survival was 94% (16/17). One death occurred among the EAP cohort that was considered by the investigator to be unrelated to OTL-103.

Across the original and integrated data sets, there were no adverse events considered to be related to OTL-103, including no evidence of oncogenesis, replication competent lentivirus or abnormal clonal proliferation. Clinical benefit was also attained in patients older than five years of age, a group considered at higher risk when treated with allogeneic hematopoietic stem cell transplantation (HSCT).

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

This presentation details the safety and efficacy of OTL-101 in 30 individuals with ADA-SCID, treated with either fresh (n=20) or cryopreserved (n=10) formulations. Patients were followed for a median of 24 months (range 12-24 months overall and 12-18 months for patients treated with the cryopreserved formulation), and results were compared with a historical cohort of 26 ADA-SCID patients treated with allogeneic hematopoietic stem cell transplantation (HSCT), including HSCT both with, and without, a matched related donor.

Results showed engraftment of genetically modified hematopoietic stem cells in 29 of 30 OTL-101 patients by six to eight months, which persisted through follow-up in both studies. Analysis of both the vector copy number in granulocytes (a measure of engraftment) and T-cell reconstitution (a relevant measure of immune recovery) showed consistent performance across the fresh and cryopreserved-treated patients.

In the OTL-101 treated patients, overall survival was 30/30 (100%) and event-free survival was 29/30 (97%). One of the 30 patients restarted treatment with enzyme replacement therapy (ERT) and subsequently withdrew from the study and received a rescue HSCT. In the historical control population, 42% of HSCT patients required re-initiation of ERT, rescue HSCT or other intervention, or died. As expected, there was no incidence of graft versus host disease in the OTL-101 group, compared with eight patients who received HSCT.

Eighteen of 20 patients (90%) in the fresh formulation study stopped immunoglobin replacement therapy (IgRT) after two years, compared to 52% of HSCT patients. Of the seven patients treated with the cryopreserved formulation with 18 months of follow-up, five had discontinued IgRT (71%), which is comparable to the 18-month data for patients treated with the fresh formulation.

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time:Monday, December 9, 7:00am ET

Investigators will present updated analyses from the ongoing proof-of-concept trial of OTL-203 for mucopolysaccharidosis type I (MPS-I).

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births inthe United Statesand between one in 200,000 and one in 1 million inEurope.3OTL-101 is an autologous,ex vivo,hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials inthe United StatesandEurope, including a registrational trial at theUniversity of California, Los Angeles(UCLA). OTL-101 has received orphan drug designation from theU.S. Food and Drug Administration(FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from theFDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is anex vivo,autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at theSan Raffaele-Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.IdTreatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is anex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele-Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard Orchard Therapeuticsis a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio ofex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by theEuropean Medicines Agencyfor severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK inApril 2018and originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italyinitiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements.Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved.Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year endedDecember 31, 2018as filed with theU.S. Securities and Exchange Commission(SEC) onMarch 22, 2019, as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency.2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314.3Kwan A, et al. JAMA. 2014;312:729-738.4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting -...

Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual…

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces presentations at the upcoming 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, Florida. The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing Process B for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

Details for Rockets poster presentations are as follows:Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor CellsSession Title: Gene Therapy and Transfer: Poster IIPresenter: Sandeep Soni, M.D.Session Date: Sunday, December 8, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene TherapySession Title: Gene Therapy and Transfer: Poster IIIPresenter: Paula Ro, Ph.D.Session Date: Monday, December 9, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Sonis presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Ro, Ph.D., Senior Scientist, Divisin de Terapias Innovadoras en el Sistema Hematopoytico, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundacin Jimnez Daz will be participating in a Q&A panel. For further information, please contact investors@rocketpharma.com.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual...