Stem Cell Clinic

Development and validation of a predictive model to guide the use of plerixafor in pediatric population | Bone Marrow Transplantation – Nature.com

The main patient characteristics are described in Table2. Type of tumor and mobilization were similar between the groups.

Based on AIC minimization, the combined variability structure was selected. The equation of the estimated base model was AP-CD34+=1.63+1.02 (PB-CD34+)+e where e had a zero-mean normal distribution N (0, s2 (k+prediction)2).

The parameter values for intercept, PB-CD34+, k, and were 1.63 (SE=0.72), 0.12 (SE=0.01), 1.76 and 0.49, respectively.

The base model showed a satisfactory goodness-of-fit plot. In the predicted vs the observed scatterplot, the dots were well scattered around the identity line indicating unbiased model predictions. Additionally, the plot of residual values confirmed non-homogeneous variance with greater residual variability for larger predicted values of the AP-CD34+ cell counts. In terms of predictive accuracy, the base model was able to properly predict the percentage of patients achieving both 2 106 and 5 106 AP-CD34+ cells/kg (Fig.2).

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, CI confidence interval.

The base model can be used to characterize the necessary counts of PB-CD34+ to achieve thresholds of 2 106 and 5 106 AP-CD34+ cells/kg (Fig.3).

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, PB-CD34+ peripheral blood-cluster of differentiation 34.

According to the base model, an estimated PB-CD34+ counts of 57.01 (90% CI: 21.76130.76) and 125.24 (90% CI: 72.09330.71) 106/L were necessary to reach thresholds of 2 106 and 5 106 AP-CD34+ cells/kg, respectively, with a probability of 0.90.

Based on AIC minimization, the best model includes the tumor type (neuroblastoma and other) as covariate. The equation of the estimated final model was as follows:

$${{{{{{{mathrm{Neuroblastoma}}}}}}}}:{{{{{{{mathrm{AP}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + = 3.01 + 0.13 times left( {{{{{{{{mathrm{PB}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + } right) + e$$

$${{{{{{{mathrm{Other}}}}}}}};{{{{{{{mathrm{tumor}}}}}}}};{{{{{{{mathrm{types}}}}}}}}:{{{{{{{mathrm{AP}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + = 0.01 + 0.13 times left( {{{{{{{{mathrm{PB}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + } right) + e$$

where e had a zero-mean normal distribution N (0, s2 (k+prediction)2).

The parameter values for intercept-neuroblastoma, intercept-other, PB-CD34+, and were 3.01 (SE=1.10), 0.01 (SE=0.006), 0.13 (SE=0.01), (simeq) 0.00 and 0.54, respectively.

According to the model, the predicted count of AP-CD34+ cells was slightly larger for neuroblastoma tumor types than for the other tumor types. It should be noted that the final model was selected considering the type of tumor as an additional covariate (in addition to PB-CD34+) based on statistical information criterion (AIC), and that the tumor type was correlated with the age of the patients - the patients with Neuroblastoma tumor type, with mean age of 3.7 years (standard deviation, SD=2.1 years), being younger than the others with mean age of 8.9 years (SD=4.8 years). However, the choice of considering tumor type in the final model instead of age was driven by the fact that the fit of the data was improved when tumor type was considered as predictor, as compared to age, which reflected in lower value of the statistical information criterion with tumor type (AIC=288.6) than with age (AIC=310.1).

The final model also showed a good predictive property in terms of goodness-of-fit plot and prediction of the percentages of patients achieving both 2 106 and 5 106 AP-CD34+ cells/kg (Fig.4). The model predicts that a smaller PB-CD34+ cell count was needed to reach 2 106 and 5 106 AP-CD34+ cells/kg with a probability of 0.90 in patients with neuroblastoma tumor type than in those with other tumor types (Fig.5). According to the final model, in patients with neuroblastoma tumor type, the estimated PB-CD34+ counts necessary to reach apheresis thresholds of 2 106 and 5 106 AP-CD34+ cells/kg with a probability of 0.90 were 27.32 (90% CI: 0.1650.51) and 103.20 (90% CI: 56.15165.18) 106/L, respectively. The estimated PB-CD34+ counts necessary to reach thresholds of 2 106 and 5 106 AP-CD34+ cells/kg with a probability of 0.90 in patients with other tumor type were 50.51 (90% CI: 29.3079.12) and 126.39 (90% CI: 77.25198.28) 106/L, respectively.

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, CI confidence interval.

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, PB-CD34+, peripheral blood-cluster of differentiation 34+.

The uncertainty related to these PB-CD34+ estimated values with the final model was slightly less in comparison to the base model probably due to a reduced residual variability.

The physiological process of stem cell mobilization via CXCR4 is comparably the same in subjects of all ages, and when adult data on CXCR4 is extrapolated into children it should closely mirror that seen in children [19]. We complemented our analyses with data from the adult NHL and MM patients who participated in the two plerixafor studies [15, 16], focusing on the first day of apheresis similar to the MOZAIC study. The details of the analyses can be found in theSupplementary section.

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CellOrigin Biotech Announces Global Strategic Collaboration with Qilu Pharma to Develop "Off-the-Shelf" CAR-iMAC Cell Therapy – BioSpace

HANGZHOU, China, Sept. 26, 2022 /PRNewswire/ -- CellOrigin Biotech (Hangzhou) Co., Ltd. announced it has made a global strategic collaboration agreement with Qilu Pharma to develop, manufacture and commercialize proprietary "off-the-shelf" induced pluripotent stem cell- (iPSC) derived Chimeric Antigen Receptor Macrophages (CAR-iMAC) for cancer immunotherapy.

The collaboration will take advantages of technologies and expertise from both parties, as well as integrate capabilities of R&D, manufacturing and marketing to develop CAR-iMAC clinical products aiming for solid tumors.

"Innovation and offering the best products that benefit patients are the core values that CellOrigin Biotech and Qilu Pharma both appreciate," said Dr. Jin Zhang, the Co-Founder of CellOrigin Biotech and a Principal Investigator of Zhejiang University, one of the top universities in China. "This is what brings us together."

"We are very excited to collaborate with Qilu Pharma because of its prestige in the field of Chinese pharmaceutical industry, as well as its tremendous track records on drug development," said Dr. Jiansong Tong, the Chief Executive Officer at CellOrigin Biotech. "Meanwhile, we will continue to seek other potential collaborators to jointly develop our innovative anti-tumor CAR-iMac cell products."

"CellOrigin Biotech is a startup company established by a group of outstanding scientists who have tremendous experiences both in R&D research and cGMP manufacture. It focused on developing innovative technologies in cell therapy and building valuable pipeline of products. It is an ideal strategic partner for novel cell therapy, and it is our pleasure to collaborate with such a great biotech company," said Qilu Pharma.

About CellOrigin Biotech

CellOrigin Biotech (Hangzhou) Co., Ltd. is dedicated to the development of genetically engineered pluripotent stem cell- (iPSC) derived immune cell therapies (such as macrophages, NK cells), with its own proprietary intellectual property. The founders are experienced leaders from Zhejiang University, Harvard University and top pharmaceutical and biotech companies around the world. They apply cutting-edge technologies in editing and differentiating iPSCs to immune cells in order to deliver novel allogeneic off-the-shelf cell therapies for the treatment of cancer patients around the world.

About Qilu Pharma

Qilu Pharma is a leading vertically integrated pharmaceutical companies in China focused on discovering, developing, manufacturing and commercializing innovative medicines. With a diverse pipeline of noval therapeutics, 10 manufacturing sites and more than 36,000 employees worldwide, Qilu is dedicated to transforming scientific innovation by internal R&D across 5 R&D platforms based in the US (Seattle WA, Boston MA, San Francisco CA) and China (Shanghai, Jinan), and external partnership globally into healthcare solutions to address unmet medical needs. To date, Qilu has launched 200+ products with 30+ products "First to launch" in China and 3 products "D181 launch" in US with approximately US$5.2 billion sales revenue in 2021. For more information, please visit http:en.qilu-pharma.com.

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University of Washington joins industry-academia alliance to accelerate research in neuroscience – EurekAlert

image:Microscopy image showing the cytoskeleton within neurons, which are differentiating from induced pluripotent stem cells. view more

Credit: UC San Francisco

The University of Washington has joined the Alliance for Therapies in Neuroscience (ATN), a long-term research partnership between academia and industry geared to transform the fight against brain diseases and disorders of the central nervous system.

Launched in 2021 by the University of California, San Francisco, UC Berkeley, Genentech a member of the Roche group and Roche Holding AG, the ATN seeks to accelerate the development of new therapies for a broad range of brain and central nervous system conditions, such as Alzheimers disease, Lou Gehrigs disease, Huntingtons disease, Parkinsons disease, autism, depression and psychiatric disorders. As part of the ATN, Genentech and Roche committed up to $53 million over 10 years for research at the ATNs participating academic institutions, a collaboration that is unique for both its duration and the breadth of its ambitions.

The Alliance for Therapies in Neuroscience is a new and transformative template for research and academia to partner, and it is an ideal collaboration for the University of Washington, said Tom Daniel, the Emeritus Joan and Richard Komen Endowed Chair and professor of biology at the UW and incoming CEO of the Washington Research Foundation, who led efforts to join the ATN. Scientists at the UW will be integrated with academic and industry partners in a way that has simply never been done before. And the UW will bring its cross-disciplinary strengths and expertise in neuroscience which span medicine, engineering and basic and clinical research to address the urgent need for new therapies, remedies and treatments in neurological diseases and disorders.

The new alliance builds on an existing academic partnership. In 2019, the UW, UCSF and UC Berkeley formed the Weill Neurohub, a $106-million, multidisciplinary endeavor supported by the Weill Family Foundation to speed discovery and innovation across neurological and psychiatric disorders, including basic research, technology development and patient care. With the UWs accession to the ATN, scientists at all three Weill Neurohub institutions can now access this novel pipeline to channel academic discoveries toward new therapies and treatments.

Pairing academic researchers with industry partners early in the research process will accelerate the transformation of academic research into clinical applications, said Dr. Stephen Hauser, the Robert A. Fishman Distinguished Professor of Neurology at UCSF and director of the UCSF Weill Institute for Neurosciences. And this long-term, 10-year commitment from Genentech and Roche means that researchers at UCSF, UC Berkeley and now the UW will benefit from years-long, close collaborations with industry. It is a type of partnership that hasnt been seen before in academic or industry research.

Membership of the UW in ATN fully leverages the vision that we and the Weills have for the Weill Neurohub, said Ehud Isacoff, the Evan Rausch Chair in Neuroscience at UC Berkeley and director of the Berkeley Brain Initiative. This collaboration with Roche and Genentech world leaders in pharma and biotech opens powerful new directions for Weill Neurohub researchers, with crucial resources and proven track records of bringing new treatments to patients and families.

Teams of scientists at ATN institutions will drive efforts to profile the progression of disease, identify new targets for therapies and model their effectiveness. Existing organizational infrastructure within the Weill Neurohub will serve to coordinate the expanded ATN efforts. In addition to Daniel, the other UW leader within the Weill Neurohub and the ATN is Dr. Jrgen Untzer, professor and chair of the Department of Psychiatry & Behavioral Sciences.

ATN endeavors are intended to meet current demands in neurological disease research and treatment, as well as lay the groundwork for future innovations in understanding and treating nervous system disorders.

The ATN is focusing on pressing needs in neurological disorders across the board: not just therapies to treat conditions like Alzheimers or Parkinsons, but also methods to diagnose them at early stages, as well as understand them at the cellular and molecular level, said Jon-Eric VanLeeuwen, director of strategic initiatives at the UCSF Weill Institute for Neurosciences.

The UW brings a variety of strengths to the ATN, according to Daniel. Neuroscience expertise at the UW spans clinical trials, cell and molecular studies, computational modeling and even research into artificial intelligence. Neuroscientists are based across the UWs STEM schools and colleges, including the School of Medicine, the College of Arts & Sciences and the College of Engineering. UW researchers have a strong track record of innovative cross-institutional collaborations in neuroscience with scientists across the region, including at the Fred Hutchinson Cancer Center and the Allen Institute, which they can also draw on for ATN research.

Through the ATN, all partners will bring their best and brightest to bear on these boiling hot, challenging problems in neuroscience, said Daniel.

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For more information, contact Daniel at danielt@uw.edu and VanLeeuwen at Jon-Eric.VanLeeuwen@ucsf.edu.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Citius Pharmaceuticals Announces a Clinical Collaboration with the University of Pittsburgh to Evaluate T-reg Cell Depletion with I/ONTAK (E7777) in…

I/ONTAK plus PD-1 checkpoint inhibitor pembrolizumab to be evaluated in solid tumor patients

University of Pittsburgh dose-ranging study expected to begin in the fourth quarter of 2022

Collaboration marks second investigator-initiated study of I/ONTAK as combination immunotherapy

CRANFORD, N.J., Sept. 22, 2022 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company developing and commercializing first-in-class critical care products,today announced a collaboration with Dr. Haider Mahdi at the University of Pittsburgh in an investigator-initiated trial to evaluate I/ONTAK ("denileukin diftitox" or "E7777") in combination with pembrolizumab in the treatment of recurrent or metastatic solid tumors.

"We are honored to support Dr. Mahdi and his team at the University of Pittsburgh in this Phase 1 investigator-initiated study to evaluate I/ONTAK as a combination therapy in the treatment of solid tumors. This study will expand the body of knowledge about I/ONTAK's unique mechanism-of-action targeting the CD25 component of the IL-2 receptor which is present on both malignant T-cells (T-cell leukemias and lymphomas) and immunosuppressive regulatory T-cells (T-regs)," stated Dr. Myron Czuczman, Chief Medical Officer of Citius. "Preclinical research in a syngeneic solid tumor mouse model shows that E7777 (denileukin diftitox) enhances anti-tumor activity and significantly extends survival benefit of anti-PD-1 therapy. This data provides a positive signal of denileukin diftitox's potential in the immuno-oncology space. There remains a significant ongoing need for innovative, effective, and well-tolerated treatments for cancer patients with solid tumors, and we are excited that I/ONTAK may provide meaningful antitumor activity in combination with the PD-1 inhibitor pembrolizumab (KEYTRUDA)," added Dr. Czuczman.

"Encouraging clinical data emerging in the field of tumor immunotherapy have demonstrated that therapies focused on enhancing T-cell responses against cancer result in a significant survival benefit in patients with advanced malignancies. Overexpression of PD-L1 on tumor cells has been reported to impede anti-tumor immunity, resulting in immune evasion. The interruption of the PD-1:PD-L1 pathway combined with diminishing the suppressive effect by T-regs may represent an attractive strategy for restoring tumor-specific T-cell immunity. This first in human I/ONTAK plus anti-PD-1 combination immunotherapy study is a significant step towards advancing a T-cell-based therapeutic approach to treating solid tumors," stated Dr. Haider Mahdi, University of Pittsburgh, Assistant Professor, Department of Obstetrics, Gynecology & Reproductive Sciences. Education & Training.

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Additionally, Citius is collaborating with an investigator-initiated study at the University of Minnesota (UMN). This Phase 1 dose-finding study to evaluate I/ONTAK prior to tisagenleucel (KYMRIAH) CAR-T therapy in patients with diffuse large B-cell lymphoma (DLBCL) enrolled its first patient in May 2021.

About the University of PittsburghStudy

The investigator-initiated trial at UPMC is an open label, Phase I/Ib study to evaluate T-reg cell depletion with I/ONTAK (E7777) in combination with pembrolizumab in recurrent or metastatic solid tumors. The study consists of two parts. Part I is a dose escalation study of four cohorts (3,6,9,12 mcg of I/ONTAK) and is expected to enroll 18-30 patients. Part II is a dose expansion study of approximately 40 patients to evaluate the safety and tolerability of the recommended combination dose of I/ONTAK and pembrolizumab. The study will also investigate the alteration of the immune microenvironment within tumors and peripheral blood. Secondary endpoints include the objective response (complete response plus partial response), progression-free survival, and overall survival.

About I/ONTAK

I/ONTAK is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. I/ONTAK, a purified version of denileukin diftitox, is a reformulation of previously FDA-approved oncology treatment ONTAK. ONTAK was marketed in the U.S. from 1999 to 2014, when it was voluntarily withdrawn from the market. Manufacturing improvements resulted in a new formulation, which maintains the same amino acid sequence but features improved purity and bioactivity. The new formulation received regulatory approval in Japan in 2021 for the treatment of CTCL and PTCL. In 2011 and 2013, the FDA granted orphan drug designation (ODD) to I/ONTAK for the treatment of PTCL and CTCL, respectively.

About Citius Pharmaceuticals, Inc.

Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, with a focus on oncology, anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapies. The Company's diversified pipeline includes two late-stage product candidates, Mino-Lok, an antibiotic lock solution for the treatment of patients with catheter-related bloodstream infections (CRBSIs), which is currently enrolling patients in a Phase 3 Pivotal superiority trial, and I/ONTAK (E7777), a novel IL-2R immunotherapy for an initial indication in cutaneous T-cell lymphoma (CTCL), for which a BLA submission is being prepared for the second half of 2022. Mino-Lokwas granted Fast Track designation by the U.S. Food and Drug Administration (FDA). I/ONTAK has received orphan drug designation by the FDA for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). In the first half of 2022, Citius initiated a Phase 2b trial for Halo-Lido, a topical formulation for the relief of hemorrhoids. The Company anticipates completing enrollment in the Halo-Lido trial by the end of 2022. For more information, please visit http://www.citiuspharma.com.

Safe Harbor

This press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "believe," "anticipate," "estimate," "expect," "plan," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: risks relating to the results of research and development activities, including those from existing and new pipeline assets; uncertainties relating to preclinical and clinical testing; our ability to successfully undertake and complete clinical trials and the results from those trials for our product candidates; our ability to commercialize our products if approved by the FDA; the estimated markets for our product candidates and the acceptance thereof by any market; the ability of our product candidates to impact the quality of life of our target patient populations; our need for substantial additional funds; the early stage of products under development; our ability to attract, integrate, and retain key personnel; our dependence on third-party suppliers; market and other conditions; risks related to our growth strategy; patent and intellectual property matters; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; our ability to procure cGMP commercial-scale supply; government regulation; competition; as well as other risks described in our SEC filings. These risks have been and may be further impacted by Covid-19. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings which are available on the SEC's website at http://www.sec.gov, including in our Annual Report on Form 10-K for the year ended September 30, 2021, filed with the SEC on December 15, 2021 and updated by our subsequent filings with the SEC. These forward-looking statements speak only as of the date hereof, and we expressly disclaim 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 or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Investor Contact:

Ilanit Allen ir@citiuspharma.com 908-967-6677 x113

Media Contact:

STiR-communications Greg Salsburg Greg@STiR-communications.com

Citius Pharmaceuticals, a late-stage biopharmaceutical company (PRNewsfoto/Citius Pharmaceuticals, Inc.)

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The power of a strong doctor-patient partnership and positivity when fighting lymphoma – Curetoday.com

After hearing the words you have cancer, a persons life instantly changes. Once the initial shock wears off, patients and their caregivers are often met with significant fear and uncertainty for the future. For Louise, a mother of five and grandmother to many more, after learning she had cancer, her oncologist, Dr. Ruemu Birhiray, MD, Hematology Oncology of Indiana, quickly assured her that they were going to fight her disease together. They adopted the motto for every problem, there is a solution, which would become an important mantra for Louise throughout her cancer journey.

Louises cancer story began in November 2019 when she went for a routine check-up. The doctors took her vitals and quickly noticed her pulse was high. Urgently, they recommended Louise go to the emergency room. Once there, Louise was given a computed tomography (CT or CAT) scan and informed that she needed to see an oncologist.

That oncologist turned out to be Dr. Birhiray, who diagnosed Louise with diffuse large B-cell lymphoma (DLBCL), the most common type of non-Hodgkin lymphoma (NHL), which affects approximately 28,000 people per year in the United States. DLBCL is a fast-growing but treatable cancer affecting B-lymphocytes, also known as B cells, a type of white blood cell that helps the body fight infections. As they develop, cancerous B cells become larger than normal and multiply uncontrollably.

Meeting Dr. Birhiray, I realized pretty quickly that he was going to take good care of me, said Louise. One of the first things you notice with Dr. Birhiray is how warm of a person he is. It felt like he was talking to me, not at me, and he was very attentive to my needs and questions.

After going through an intensive chemotherapy regimen, Louise was thrilled to hear that her DLBCL had gone into remission, only to learn six months later that the cancer had returned. That unfortunately made Louise one of up to 50% of patients whose DLBCL relapses (returns) or does not respond to treatment (becomes refractory).

While initially devasted to hear the cancer had returned, Louise and Dr. Birhiray swiftly worked together to determine next steps.

When Louises cancer relapsed after initial treatment, I wanted to take a different approach, said Dr. Birhiray. For her next treatment, I initially thought about doing a bone marrow transplant, but together we decided it wasnt appropriate. So I considered a different treatment regimen that had proven results in certain patients and did not require a hospital stay.

Dr. Birhiray again assured Louise that for every challenge, there are options. For Louise, that option was ultimately Monjuvi (tafasitamab-cxix) a targeted immunotherapy treatment given with another medicine called lenalidomide to treat adults with certain types of DLBCL that has come back or that did not respond to previous treatment and who cannot receive a stem cell transplant.

Shortly after beginning treatment with Monjuvi and lenalidomide, Louise had to go to another unrelated check-up that involved a CAT scan, where she received extremely encouraging news: her cancer was showing rapid improvement. A couple of months later, a positron emission tomography (PET) scan revealed there were no detectable signs of cancer in her body.

It was such a relief to no longer see any cancer on the CAT scan and to have confirmation that treatment with Monjuvi had worked for me, said Louise. Im very grateful to Dr. Birhiray and my healthcare team for helping me through my journey with DLBCL so far. They are always so positive and honest with me, and I feel like they listened to my needs and included me in important decisions, which made getting through treatment so much easier.

Louise is representative of a patient responding to Monjuvi. Every patient is different and individual results may vary. Louise continues to be under the care of Dr. Birhiray who routinely checks to make sure her cancer has not returned. Please read the Important Safety Information below to learn more about the side effects of Monjuvi.

Dr. Birhiray was also pleased by Louises results with Monjuvi. From their very first meeting, he had worked closely with Louise to develop a treatment plan that took into account her individual needs and circumstances.

According to Dr. Birhiray, it is also critical to involve patients in treatment-related decisions by having open and honest conversations about their options and what will happen in their body with certain treatments, including possible side effects. Beyond the effects of treatments, Dr. Birhiray believes it is important to account for a patients treatment goals, which includes factors such as the experience they are seeking while undergoing treatment and their life goals while on and following treatment.

As an oncologist, you really need to get to know your patient and establish trust from the beginning said Dr. Birhiray. More than just knowing their names, you need to know their bodies and understand their disease and how it affects them in order to develop a treatment plan that meets their needs. This requires in-depth conversations that can take upwards of an hour, in which I ensure my patients are knowledgeable about the treatment they are about to undergo and how it will affect them, and to understand how they are feeling.

Louises DLBCL continues to be in remission and she has a positive outlook for her future. She hopes her story can inspire others to build relationships with their doctors like the one she has with Dr. Birhiray.

I feel extremely fortunate to have found Dr. Birhiray and received a treatment for my DLBCL that led to remission, said Louise. I feel strongly that my outcome is a result of the partnership with my oncologist, working together to come up with a treatment plan that was right for me.

If you, like Louise, have DLBCL that came back or didnt respond to the first treatment (relapsed or refractory DLBCL), start a conversation with your healthcare team about your options. To learn more about Monjuvi, DLBCL and for support and resources, visit http://www.Monjuvi.com.

What is MONJUVI?

MONJUVI (tafasitamab-cxix) is a prescription medicine given with lenalidomide to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL) that has come back (relapsed) or that did not respond to previous treatment (refractory) and who cannot receive a stem cell transplant.

It is not known if MONJUVI is safe and effective in children.

The approval of MONJUVI is based on a type of response rate. There is an ongoing study to confirm the clinical benefit of MONJUVI.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of MONJUVI?

MONJUVI may cause serious side effects, including

The most common side effects of MONJUVI include

These are not all the possible side effects of MONJUVI. Your healthcare provider will give you medicines before each infusion to decrease your chance of infusion reactions. If you do not have any reactions, your healthcare provider may decide that you do not need these medicines with later infusions. Your healthcare provider may need to delay or completely stop treatment with MONJUVI if you have severe side effects.

Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you

You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

Tell your healthcare provider about all the medications you take, including prescription and over- the-counter medicines, vitamins, and herbal supplements.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. You may also report side effects to MORPHOSYS US INC. at (844) 667-1992.

DIRECTIONALS TO THE PI:

Please see the full Prescribing Information, including Patient Information, for additional Important Safety Information.

These participants were compensated for their time.

RC-US-TAF-01576 August 2022

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AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AVR-RD-04, an investigational gene therapy for the treatment of cystinosis, a life-threatening disease that causes progressive multi-organ damage, including early, acute kidney disease progressing to end-stage kidney disease.

FDAs Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.

AVR-RD-04 is designed to genetically modify patients own hematopoietic stem cells (HSCs) to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis.

Preliminary data from the ongoing University of California San Diego Phase 1/2 clinical trial suggest that this approach is well tolerated, with no adverse events (AEs) related to the drug product reported to date. All AEs reported were related to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions. The majority of AEs were mild or moderate and resolved without clinical sequelae. Clinical data to date indicate this investigational approach provides benefits in multiple tissues evaluated, including the eyes, skin, gastrointestinal mucosa and the neurocognitive system. The collaborator-sponsored Phase 1/2 clinical trial is funded in part by grants to University of California San Diego from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).

About Cystinosis Cystinosis is a rare, progressive disease that impacts approximately 1,600 patients in the U.S., Europe and Japan and is marked by the accumulation of cystine in cellular organelles known as lysosomes. Untreated cystinosis is fatal at an early age. The current SOC for cystinosis, a treatment regimen that can require dozens of pills per day, does not prevent overall disease progression and carries side effects, such as breath and body odor and gastrointestinal symptoms, which can impede compliance. More than 90% of treated cystinosis patients require a kidney transplant in the second or third decade of life.

About AVROBIO Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients own hematopoietic stem cells (HSCs), with the goal to durably express the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for cystinosis and Gaucher disease type 1, as well as preclinical programs for Gaucher disease type 3, Hunter syndrome and Pompe disease. Our proprietary plato gene therapy platform is designed to be scaled to support late-stage clinical development and commercialization globally. We are headquartered in Cambridge, Mass. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our preclinical and clinical product candidates, including AVR-RD-04 for the treatment of cystinosis, the potential benefits and incentives provided by FDAs rare pediatric disease designation for AVR-RD-04, the design, commencement, enrollment and timing of planned clinical trials, preclinical or clinical trial results, product approvals and regulatory pathways, our plans and expectations with respect to interactions with regulatory agencies, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our preclinical and investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today 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 risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, including beneficial effects seen in multiple organs and tissues, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis...