Category Archives: Genetic Medicine

--Conference Session: Therapeutic Applications of Extracellular Vesicles: From Diagnostics to Drug Delivery--

LOS ANGELES, May 12, 2021 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that Linda Marbn, Ph.D., Capricors CEO, will present at the American Society of Gene and Cell Therapys (ASGCT) 24th annual meeting, being held virtually May 11-14, 2021.

We are honored to have been selected as an industry leader on exosome delivery for SARS-CoV-2 vaccinations. This premier international symposium is a key medical conference in our field and brings together the leading scientists and healthcare professionals focused on gene and cell therapies, said Linda Marbn, Ph.D. chief executive officer of Capricor.

Please visit the Investor Relations section of the Company website for archived webcasts and investor materials available athttp://capricor.com/news/events/.

About ASGCT

The mission of ASGCT is to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. ASGCTs strategic vision is to be a catalyst for bringing together scientists, physicians, patient advocates, and other stakeholders to transform the practice of medicine by incorporating the use of genetic and cellular therapies to control and cure human disease.

Details for the 2021 ASGCT virtual meeting are available on the meeting website athttps://annualmeeting.asgct.org.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell- and exosome-based therapeutics and vaccines for the treatment and prevention of a broad spectrum of diseases. Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Capricor is also developing our exosomes platform technology as a next-generation therapeutic platform. Our current focus is on the development of exosomes loaded with nucleic acids, including mRNA, to treat or prevent a variety of diseases. For more information, visitwww.capricor.comand follow the Company onFacebook,InstagramandTwitter.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2020 as filed with the Securities and Exchange Commission on March 15, 2021. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricors exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Media Contact:Caitlin Kasunich / Raquel ConaKCSA Strategic Communicationsckasunich@kcsa.com/rcona@kcsa.com212.896.1241 / 212.896.1204

Investor Contact:Joyce AllaireLifeSci Advisors, LLCjallaire@lifesciadvisors.com617.435.6602

Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com310.358.3200

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Capricor to Present at the American Society of Gene and Cell Therapy's 24th Annual Meeting - GlobeNewswire

The Canadian Press

GAZA CITY, Gaza Strip (AP) Just weeks ago, the Gaza Strips feeble health system was struggling with a runaway surge of coronavirus cases. Authorities cleared out hospital operating rooms, suspended nonessential care and redeployed doctors to patients having difficulty breathing. Then, the bombs began to fall. This week's violence between Israel and Gaza's Hamas rulers has killed 103 Palestinians, including 27 children, and wounded 530 people in the impoverished territory. Israeli airstrikes have pounded apartments, blown up cars and toppled buildings. Doctors across the crowded coastal enclave are now reallocating intensive care unit beds and scrambling to keep up with a very different health crisis: treating blast and shrapnel wounds, bandaging cuts and performing amputations. Distraught relatives didn't wait for ambulances, rushing the wounded by car or on foot to Shifa Hospital, the territorys largest. Exhausted doctors hurried from patient to patient, frantically bandaging shrapnel wounds to stop the bleeding. Others gathered at the hospital morgue, waiting with stretchers to remove the bodies for burial. At the Indonesia Hospital in the northern town of Jabaliya, the clinic overflowed after bombs fell nearby. Blood was everywhere, with victims lying on the floors of hallways. Relatives crowded the ER, crying out for loved ones and cursing Israel. Before the military attacks, we had major shortages and could barely manage with the second (virus) wave, said Gaza Health Ministry official Abdelatif al-Hajj by phone as bombs thundered in the background. Now casualties are coming from all directions, really critical casualties. I fear a total collapse." Gutted by years of conflict, the impoverished health care system in the territory of more than 2 million people has always been vulnerable. Bitter division between Hamas and the West Bank-based Palestinian Authority and a nearly 14-year blockade imposed by Israel with Egypt's help also has strangled the infrastructure. There are shortages of equipment and supplies such as blood bags, surgical lamps, anesthesia and antibiotics. Personal protection gear, breathing machines and oxygen tanks remain even scarcer. Last month, Gaza's daily coronavirus cases and deaths hit record highs, fueled by the spread of a variant that first appeared in Britain, relaxation of movement restrictions during Ramadan, and deepening public apathy and intransigence. In the bomb-scarred territory where the unemployment rate is 50%, the need for personal survival often trumps the pleas of public health experts. While virus testing remains limited, the outbreak has infected more than 105,700 people, according to health authorities, and killed 976. As cases climbed last year, stirring fears of a health care catastrophe, authorities set aside clinics just for COVID-19 patients. But that changed as airstrikes pummeled the territory. Nurses at the European Hospital in the town of Khan Younis, frantically needing room for the wounded, moved dozens of virus patients in the middle of the night to a different building, said hospital director Yousef al-Akkad. Its surgeons and specialists, who had deployed elsewhere for the virus, rushed back to treat head injuries, fractures and abdominal wounds. If the conflict intensifies, the hospital won't be able to care for the virus patients, al-Akkad said. We have only 15 intensive care beds, and all I can do is pray, he said, adding that because the hospital lacks surgical supplies and expertise, hes already arranged to send one child to Egypt for reconstructive shoulder surgery. I pray these airstrikes will stop soon. At Shifa, authorities also moved the wounded into its 30 beds that had been set aside for virus patients. Thursday night was the quietest this week for the ICU, as bombs had largely fallen elsewhere in Gaza. Patients with broken bones and other wounds lay amid the din of beeping monitors, intercoms and occasional shouts by doctors. A few relatives huddled around them, recounting the chaotic barrage. About 12 people down in one airstrike. It was 6 p.m. in the street. Some were killed, including my two cousins and young sister. Its like this every day, said 22-year-old Atallah al-Masri, sitting beside his wounded brother, Ghassan. Hospital director Mohammed Abu Selmia lamented the latest series of blows to Gaza's health system. The Gaza Strip is under siege for 14 years, and the health sector is exhausted. Then comes the coronavirus pandemic, he said, adding that most of the equipment is as old as the blockade and can't be sent out for repairs. Now, his teams already strained by virus cases are treating bombing victims, more than half of whom are critical cases needing surgery. They work relentlessly, he added To make matters worse, Israeli airstrikes hit two health clinics north of Gaza City on Tuesday. The strikes wreaked havoc on Hala al-Shawa Health Center, forcing employees to evacuate, and damaged the Indonesian Hospital, according to the World Health Organization. Israel, already under pressure from an International Criminal court investigation into possible war crimes during the 2014 war, reiterated this week that it warns people living in targeted areas to flee. The airstrikes nonetheless have killed civilians and inflicted damage on Gaza's infrastructure. The violence also has closed a few dozen health centers conducting coronavirus tests, said Sacha Bootsma, director of WHO's Gaza office. This week, authorities conducted some 300 tests a day, compared with 3,000 before the fighting began. The U.N. Relief and Works Agency, or UNRWA, ordered staff to stay home from its 22 clinics for their safety. Those now-closed centers had also administered coronavirus vaccines, a precious resource in a place that waited months to receive a limited shipment from the U.N.-backed COVAX program. Those doses will expire in just a few weeks and get thrown away, with huge implications for authorities' ability to mobilize additional vaccines in the future," Bootsma said. For the newly wounded, however, the virus remains an afterthought. The last thing that Mohammad Nassar remembers before an airstrike hit was walking home with a friend on a street. When he came to, he said, we found ourselves lying on the ground. Now the 31-year-old is hooked up to a tangle of tubes and monitors in the Shifa Hospital surgical ward, with a broken right arm and a shrapnel wound in his stomach. - DeBre reported from Dubai, Untied Arab Emirates. Isabel Debre And Fares Akram, The Associated Press

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Investing in high growth stocks in the U.S. and Asia during tense times - Yahoo Canada Finance

CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces positive clinical data from its Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) gene therapy programs presented at the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

We are very excited to report positive clinical results from three of our lentiviral-based gene therapy programs at this years ASGCT, which show the great potential of these therapies to successfully treat FA, LAD-I and PKD. In the case of RP-L102 for FA and RP-L201 for LAD-I, the new data advance us closer to regulatory submissions, said Gaurav Shah, M.D., Chief Executive Officer of Rocket. At least six out of nine patients in our FA Phase 1 and 2 trials now show evidence of engraftment, further supporting the potential of RP-L102 to serve as a hematologic treatment option for FA in the absence of cytotoxic conditioning. Although preliminary, four out of the five patients anticipated necessary for a positive trial outcome have initially met the minimum 10% MMC resistance threshold in the bone marrow on at least one occasion, including two patients at 6-months post-treatment.

Dr. Shah continued, In our Phase 1/2 trial for LAD-I, all four patients with follow-up ranging from 3 to 18 months had CD18 expression that substantially exceeded the 4-10% threshold associated with survival into adulthood and consistent peripheral blood vector copy number, further demonstrating the potential of RP-L201 to yield durable clinical benefit. All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy. Lastly, data from our Phase 1 trial of RP-L301 for PKD show that both patients hemoglobin levels have safely normalized, with neither patient requiring red blood cell transfusions after hematopoietic reconstitution while demonstrating improving hemolysis markers. We are proud of the progress we have made across all three programs and look forward to further advancing our investigational gene therapies to offer curative treatments to patients with these devastating diseases.

Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical Trials

The data described in the presentation are from nine pediatric patients treated with RP-L102, Rockets ex vivo lentiviral gene therapy candidate for FA.

Presentation Details:Session: Hematologic and Immunologic DiseasesPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineDate: Tuesday, May 11, 2021Time: 8:00-10:00 a.m. EDT

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

The data presented in the oral presentation are from four pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%, who were treated with RP-L201, Rockets ex-vivo lentiviral gene therapy candidate. The safety profile of RP-L201 appears favorable with all infusions well tolerated and no drug product-related serious adverse events (SAEs) .

Preliminary efficacy was evident in all four patients, including two patients with at least 9-months of follow-up. All four patients demonstrated CD18 expression consistent with the reversal of severe LAD-I phenotype.

Most importantly, each of these patients were able to leave the hospital in the weeks following RP-L201 therapy, and all have been at home without any serious or severe infections following hospital discharge.

Presentation Details:Session: Genetic Blood and Immune DisordersPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesDate: Tuesday, May 11, 2021Time: 6:15-6:30 p.m. EDT

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients

The data presented in the oral presentation are from two adult patients with significant anemia and transfusion requirements. The patients were treated with RP-L301, Rockets ex vivo lentiviral gene therapy candidate for PKD. RP-L301 continued to be well tolerated, with no serious safety issues or infusion-related complications observed up to 9-months post-treatment.

Preliminary efficacy was evident in both patients during the initial 9-months and 3-months post-treatment, respectively.

Presentation Details:Session: Gene Therapies for HemoglobinopathiesPresenter: Jos Luis Lpez Lorenzo, M.D., Hospital Universitario Fundacin Jimnez Daz, Madrid, SpainDate: Wednesday, May 12, 2021Time: 6:45-7:00 p.m. EDT

In addition, the following presentations at this years conference also detail results from Rocket Pharma clinical studies:

Title: Gene Therapy in Fanconi Anemia: Follow-Up of a Phase I/II Gene Therapy Trial in Patients with Fanconi Anemia, Subtype ASession: Genetic Blood and Immune DisordersPresenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Tuesday, May 11, 2021Time: 5:30-5:45 a.m. EDT

Select results from Dr. Buerens presentation will also be highlighted by Paula Rio, Ph.D. Details for this Invited Presentation are as follows:

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene TherapyPresenter: Paula Ro, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday May 13, 2021Time: 10:00-11:45 a.m. EDT

Title: LV-Mediated Gene Therapy of Pyruvate Kinase DeficiencySession: Cutting Edge Gene and Cell Therapy Research in Europe (Organized by ESGCT)Presenter: Jose-Carlos Segovia, Head of the Differentiation and Cytometry Unit, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Wednesday May 12, 2021Time: 10:52-11:18 a.m. EDT

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 Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a 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 natural 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 cells.

About Leukocyte Adhesion Deficiency-I Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (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, Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. 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 its guidance for 2021 in light of COVID-19, 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 monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, 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 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, 2020, filed March 1, 2021 with the SEC. 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 Presents Positive Clinical Data from Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency Programs...

Introduction

Myopia, the most common refractive error, results in a significant threat on global public health worldwide.1 As the myopic population increases globally, the severity of its impact is predicted.2 Children with early onset are particularly susceptible to myopia-related complications, like high myopia (HM) and myopic macular degeneration.3 According to a recent school-based epidemiology study of myopia in China with 14,551 participants (ages ranging from 5 to 16 years), the overall prevalence of myopia is 78.2%.4 Myopia is a complex disease which is contributed by various environmental and genetic factors. Environmental factors includes low outdoor time and near work, dim light exposure, the use of LED lamps for homework, low sleeping hours, and short reading distance.3,5 Meanwhile, there is growing evidence that susceptibility genes play a crucial role in the risk of myopia and single nucleotide polymorphisms (SNPs) may contribute to the risk of myopia.610

Recently, a genome-wide association study (GWAS) identified six novel loci (rs2246661, rs74633073, rs76903431, rs698047, rs17029206, and rs72748160) in Asian adults, and revealed the important role of genes in the nervous system in the pathogenesis of myopia.11 The findings highlighted a nervous system role in pathogenesis of myopia. Minors are better suited to studying the genetic factors of myopia. Whether these genetic loci works for myopia in minors remains unknown and worthy to be explored. Thus, here we aimed to evaluate the potential role of these GWAS identified loci in occurrence of myopia in this case-control study including 600 myopia minors, 110 HM minors and 800 non-myopia minors.

A total of 600 consecutive myopia minors, 110 HM minors, and 800 non-myopia minors, which were frequency-matched by age and gender, were recruited in this case-control study. All subjects were Chinese Han population. Myopia was defined as mean spherical equivalent (MSE) of both eyes 0.5 diopters (D), while HM was defined as MSE less than or equal to 6.0 D.12 Patients with a predisposition to myopic eye disease, other known ocular or systemic diseases were excluded. Controls were selected from subjects coming for routine vision screening. The criteria for the control group were as follows: minors with MSE between 0.5 D and +1.0 D, best unaided visual acuity 0.8, and no other known ocular or systemic diseases.13 Patients are tested for refractive errors using an automated refractometer (Topcon RM-8000B, Topcon Co., Tokyo, Japan). The refraction was taken under cycloplegia. Information of all participants, including age, gender, body mass index (BMI, calculated using weight/height2), self-reported outdoors time, self-reported time using electronic equipment, and parental myopia, was collected through questionnaire responses, and all subjects donated 5 mL peripheral venous blood. The study protocol was approved by the ethics committee of Nanjing Tongren Hospital. All subjects gave their written informed consent, and the study complied with the Declaration of Helsinki.

Genomic DNA was extracted by blood DNA extraction kit (Promega, Madison, Wisconsin, USA) and stored in TE buffer. Genotyping was performed by TaqMan assay in 384-well ABI 7900HT Real-Time PCR system (Applied Biosystems [ABI], Foster City, CA). The qPCR reactions proceeded in a final volume of 10 L mix including 5 L TaqMan Genotyping Master Mix (Thermo Fisher Scientific), 0.5 L pre-designed TaqMan probe (Thermo Fisher Scientific), 20 ng genomic DNA and ultrapure water. Each plate included blank samples as negative controls to verify genotyping quality. Genotype data were analyzed using their System SDS Allelic Discrimination Software version 2.3 (Applied Biosystems). For quality control, about 5% of the samples were genotyped repeatedly with Sanger sequencing and the results of both methods were in good agreement.

SPSS 22.0 (SPSS, Chicago, IL) was used for statistical analysis, and a two-sided P-value of less than 0.05 was used as statistical significance. Chi -square goodness-of-fit test was adopted to derive the Hardy-Weinberg equilibrium (HWE). In the case-control study, Student t-test and/or Chi -square test were used to demonstrate how demographic and clinical characteristics and frequency of genotypes differ between case and control groups. Using unconditional logistic regression model, adjusted odds ratios (ORs) and 95% confidence intervals (CIs) were adopted (only significant variables in Table 1 were included for adjustment) to evaluate the effects of SNPs and to quantify the association between the SNPs and myopia in minors.

Table 1 Characteristics of Participating Minors

Table 1 presented the 600 myopia minors, 110 HM minors, and 800 non- myopia minors in this case-control study. The groups were comparable in age, and gender (P>0.05). While the highly myopic spent more time using electronic devices (P<0.001), less time outdoors (P<0.001) and had more myopic parents than non-myopic ones (P=0.015). The median ages in controls, myopia, and HM were 15.1, 15.0, and 15.1, respectively, while the means standard deviation for them were 0.320.41, 3.2 1.6, and 9.82.2, respectively.

All six SNPs analyzed were in HWE in non-myopia controls, indicating that the sampled subjects were representative of the population and did not show any bias in genotype frequency (p>0.05). Subsequently, we evaluated the associations between the selected SNPs and the risk of myopia adjusting for BMI, self-reported outdoors time, and self-reported time using electronic equipment. Table 2 showed the results of genotypic frequency analysis for selected loci. SNP rs2246661 (allelic OR: 1.29; 95% CI: 1.091.52; P =0.003), rs74633073 (allelic OR: 1.41; 95% CI: 1.121.78; P =0.004), and rs76903431 (allelic OR: 1.42; 95% CI: 1.111.81; P =0.005) significantly contributed to elevated susceptibility of myopia. Under additive genetic model, all of three SNPs showed statistically significant associations. For rs2246661, the CT genotype was associated with a 1.42-fold increased risk (95% CI= 1.121.81; P=0.004), while the TT genotype conferred 1.64-fold increased risk of myopia (95% CI= 1.12.43; P=0.014), compared with the TT genotype. For rs74633073, the CT genotype was associated with a 1.39-fold increased risk (95% CI= 1.051.85; P=0.022), while the TT genotype conferred 2.61-fold increased risk of myopia (95% CI= 1.126.08; P=0.026). For rs76903431, genotype GG was associated with a 1.48-fold increased risk (95% CI= 1.111.97; P=0.007), while the GG genotype conferred 2.57-fold increased risk of myopia (95% CI= 1.046.37; P=0.041), compared with the CC genotype.

Table 2 Associations Between Candidate Loci and Myopia in Minors

We further evaluated the associations of these six candidate SNPs with HM adjusting for self-reported outdoors time, self-reported time using electronic equipment, and parental myopia. We only found rs2246661 (OR: 1.37; 95% CI: 1.021.84; P =0.035), significantly contributed to elevated susceptibility of HM (Table 3). Under additive genetic model, the CT genotype was associated with a 1.55-fold increased risk (95% CI= 1.012.37; P=0.044), while the TT genotype conferred 1.88-fold increased risk of myopia (95% CI=1.033.43; P=0.040), compared with the TT genotype.

Table 3 Associations Between Candidate Loci and High Myopia in Minors

The current study investigated the potential function of six GWAS identified loci in occurrence of minors' myopia in a case-control study in Chinese population. We found three loci, including rs2246661, rs74633073, and rs76903431, significantly contributed to elevated risk of myopia. Besides, we also found rs2246661 significantly contributed to HM in minors. Our results confirm the GWAS findings in Asian adults and further provide a causal explanation for the occurrence of myopia at the molecular level.

The prevalence of myopia grew rapidly in minors.3,5,14 Finding the causes of the disease and taking effective preventive measures are vital to controlling the damage caused by myopia in young people. To date, a series of GWASs have been conducted to characterize the molecular mechanism responsible for myopia worldwide.11, 1520 However, not all could be replicated. For example, Wang et al21 replicated findings of two Japanese GWAS in a Chinese population, and got null results. This was because myopia in adults was a genetically heterogeneous disease, which was influenced by inborn genetic factors and acquired environmental factors. On the contrary, minors are better suited to exploring the genetic factors of myopia. Thus, we attempted to classify the occurrence of myopia in minors was affected by GWAS loci identified in adults in this case-control study.

In the current study, rs2246661, rs74633073, and rs76903431 were identified to be associated risk of myopia in minors. Through searching Pubmed, we did not find any other genetic associations. According to RegulomeDB 2.0, rs2246661 and rs74633073 were located at the transcription factor (TF) binding site, which could affect the combination of TFs and their targets.22 HaploReg v4.1 revealed rs2246661 could cause Ets Motifs change, and rs74633073 could cause AP-2, RFX5 Motifs change, while rs76903431 could cause CDP, Pbx-1, RXRA Motifs change.23 This evidence supported the important role of these genetic loci.

Our study had several limitations. First, the selection bias of a case-control study design cannot be avoided. Second, early-onset myopia, which refers to myopia occurring before the age of 11 years, was not evaluated in current study, due to the limitations of sample size.24 Third, based on existing sample size, the associations might not have the strength to achieve real results, especially for HM. Fourth, the biological function of these SNPs and its detailed effect on occurrence of myopia need to be deep investigated by further biological studies. There are also several strengths in our research, including the detailed inspection and accurate diagnosis of cases, structured questionnaire by well-trained interviewers, and strict quality control of genotyping.

Conclusively, this study provides the evidence of the promotional role of rs2246661, rs74633073, and rs76903431 loci on the susceptibility of myopia. Replicated researches in independent ethnic samples and functional investigation are needed to confirm our findings.

The authors declare that they have no conflict of interest.

1. Foster PJ, Jiang Y. Epidemiology of myopia. Eye (Lond). 2014;28(2):202208. doi:10.1038/eye.2013.280

2. Angle J, Wissmann DA. The epidemiology of myopia. Am J Epidemiol. 1980;111(2):220228. doi:10.1093/oxfordjournals.aje.a112889

3. Grzybowski A, Kanclerz P, Tsubota K, Lanca C, Saw SM. A review on the epidemiology of myopia in school children worldwide. BMC Ophthalmol. 2020;20(1):27. doi:10.1186/s12886-019-1220-0

4. Wang J, Li Y, Zhao Z, et al. School-based epidemiology study of myopia in Tianjin, China. Int Ophthalmol. 2020;40(9):22132222. doi:10.1007/s10792-020-01400-w

5. Mak CY, Yam JC, Chen LJ, Lee SM, Young AL. Epidemiology of myopia and prevention of myopia progression in children in East Asia: a review. Hong Kong Med J. 2018;24(6):602609.

6. Kunceviciene E, Liutkeviciene R, Budiene B, Sriubiene M, Smalinskiene A. Independent association of whole blood miR-328 expression and polymorphism at 3UTR of the PAX6 gene with myopia. Gene. 2019;687:151155. doi:10.1016/j.gene.2018.11.030

7. Zhang D, Zeng G, Hu J, McCormick K, Shi Y, Gong B. Association of IGF1 polymorphism rs6214 with high myopia: a systematic review and meta-analysis. Ophthalmic Genet. 2017;38(5):434439. doi:10.1080/13816810.2016.1253105

8. Jin GM, Zhao XJ, Chen AM, Chen YX, Li Q. Association of COL1A1 polymorphism with high myopia: a Meta-analysis. Int J Ophthalmol. 2016;9(4):604609.

9. Liang Y, Song Y, Zhang F, Sun M, Wang N. Effect of a single nucleotide polymorphism in the LAMA1 promoter region on Transcriptional activity: implication for pathological myopia. Curr Eye Res. 2016;41(10):13791386. doi:10.3109/02713683.2015.1118129

10. Chen T, Shan G, Ma J, Zhong Y. Polymorphism in the RASGRF1 gene with high myopia: a meta-analysis. Mol Vis. 2015;21:12721280.

11. Meguro A, Yamane T, Takeuchi M, et al. Genome-wide association study in asians identifies novel loci for high myopia and highlights a nervous system role in its pathogenesis. Ophthalmology. 2020;127(12):16121624. doi:10.1016/j.ophtha.2020.05.014

12. Luo HD, Gazzard G, Liang Y, Shankar A, Tan DT, Saw SM. Defining myopia using refractive error and uncorrected logMAR visual acuity >0.3 from 1334 Singapore school children ages 79 years. Br J Ophthalmol. 2006;90(3):362366. doi:10.1136/bjo.2005.079657

13. Tideman JW, Polling JR, Voortman T, et al. Low serum vitamin D is associated with axial length and risk of myopia in young children. Eur J Epidemiol. 2016;31(5):491499. doi:10.1007/s10654-016-0128-8

14. Recko M, Stahl ED. Childhood myopia: epidemiology, risk factors, and prevention. Mo Med. 2015;112(2):116121.

15. Huang Y, Kee CS, Hocking PM, et al. A genome-wide association study for susceptibility to visual experience-induced myopia. Invest Ophthalmol Vis Sci. 2019;60(2):559569. doi:10.1167/iovs.18-25597

16. Khor CC, Miyake M, Chen LJ, et al. Genome-wide association study identifies ZFHX1B as a susceptibility locus for severe myopia. Hum Mol Genet. 2013;22(25):52885294. doi:10.1093/hmg/ddt385

17. Meng W, Butterworth J, Bradley DT, et al. A genome-wide association study provides evidence for association of chromosome 8p23 (MYP10) and 10q21.1 (MYP15) with high myopia in the French Population. Invest Ophthalmol Vis Sci. 2012;53(13):79837988. doi:10.1167/iovs.12-10409

18. Li Z, Qu J, Xu X, et al. A genome-wide association study reveals association between common variants in an intergenic region of 4q25 and high-grade myopia in the Chinese Han population. Hum Mol Genet. 2011;20(14):28612868. doi:10.1093/hmg/ddr169

19. Solouki AM, Verhoeven VJ, van Duijn CM, et al. A genome-wide association study identifies a susceptibility locus for refractive errors and myopia at 15q14. Nat Genet. 2010;42(10):897901. doi:10.1038/ng.663

20. Hysi PG, Young TL, Mackey DA, et al. A genome-wide association study for myopia and refractive error identifies a susceptibility locus at 15q25. Nat Genet. 2010;42(10):902905. doi:10.1038/ng.664

21. Wang Q, Gao Y, Wang P, et al. Replication study of significant single nucleotide polymorphisms associated with myopia from two genome-wide association studies. Mol Vis. 2011;17:32903299.

22. Boyle AP, Hong EL, Hariharan M, et al. Annotation of functional variation in personal genomes using Regulome DB. Genome Res. 2012;22(9):17901797. doi:10.1101/gr.137323.112

23. Ward LD, Kellis M. HaploReg v4: systematic mining of putative causal variants, cell types, regulators and target genes for human complex traits and disease. Nucleic Acids Res. 2016;44(D1):D877881. doi:10.1093/nar/gkv1340

24. Baird PN, Saw SM, Lanca C, et al. Myopia. Nat Rev Dis Primers. 2020;6(1):99.

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Genetic association study revealed three novel loci were ass | PGPM - Dove Medical Press

Russian Geographical Society

When you hold a job like Defense Minister of Russia, you presumably have to be bold and think outside the box to protect your country from enemy advances. And with his latest strategic ideacloning an entire army of ancient warriorsSergei Shoigu is certainly taking a big swing.

In an online session of the Russian Geographical Society last month, Shoigu, a close ally of Russian President Vladimir Putin, suggested using the DNA of 3,000-year-old Scythian warriors to potentially bring them back to life. Yes, really.

First, some background: The Scythian people, who originally came from modern-day Iran, were nomads who traveled around Eurasia between the 9th and 2nd centuries B.C., building a powerful empire that endured for several centuries before finally being phased out by competitors. Two decades ago, archaeologists uncovered the well-preserved remains of the soldiers in a kurgan, or burial mound, in the Tuva region of Siberia.

Because of Tuvas position in southern Siberia, much of it is permafrost, meaning a form of soil or turf that always remains frozen. Its here where the Scythian warrior saga grows complex, because the frozen soil preserves biological matter better than other kinds of ground. Russian defense minister Sergei Shoigu knows this better than anyone, because hes from Tuva.

Of course, we would like very much to find the organic matter and I believe you understand what would follow that, Shoigu told the Russian Geographical Society. It would be possible to make something of it, if not Dolly the Sheep. In general, it will be very interesting.

Shoigu subtly suggested going through some kind of human cloning process. But is that even possible?

To date, no one has cloned a human being. But scientists have successfully executed the therapeutic cloning of individual kinds of cells and other specific gene-editing work, and of course, there are high-profile examples of cloning pretty complex animals. Earlier this year, for example, scientists cloned an endangered U.S. species for the first time: a black-footed ferret whose donor has been dead for more than 30 years.

So, why are humans still off the menu?

Blame a technical problem with the most common form of cloning, which is called nuclear transfer. In this process, a somatic cell (like a skin or organ cell, with a specific established purpose in the body) has its nucleus carefully lifted out, and this nucleus is deposited in an oocyte, or egg cell, with its nucleus carefully removed. Its like a blank template waiting to have a new nucleus swapped in.

gremlinGetty Images

From a technical perspective, cloning humans and other primates is more difficult than in other mammals, the National Institutes of Healths (NIH) National Human Genome Research Institute says on its website:

You might remember spindle proteins from your mitosis diagrams back in high school biology. And while theres a relatively easy way around this problem, its almost moot when cloning humans is considered extremely taboo in most of the world. In some places, its also explicitly illegal.

We would like very much to find the organic matter and I believe you understand what would follow that.

Curiously, the U.S. hasnt banned the gene editing of embryos. But the NIH doesnt fund research on the practice, and places like in-vitro clinics arent allowed to do any non-U.S. Food and Drug Administration-approved manipulation of embryos under any circumstances.

That example starts to illustrate why the problem is so complexbecause a lot of cutting-edge genetic medicine is walking right up to the line without crossing it. Making laws that address full human embryo cloning, then, requires a jigsaw puzzle of careful language that doesnt rule out these kinds of therapeutic cloning.

HandoutGetty Images

But lets say Russia ignores all legality in favor of Shoigus big plans. In that case, scientists would have to develop a way to lift out the human nucleus without damaging the cell beyond repair.

Scientists have cloned certain monkeys, so primates are at least hypothetically still in the mix, despite the spindle proteins. But the success rate even for non-primate clones is already very lowit took Dolly the sheeps research team 277 attempts to get a viable embryo.

And what if all of that went perfectly? Well, the Scythians were powerful warriors and gifted horsemen, but scientistsor the Kremlinmust carefully monitor a cloned baby version of a deceased adult warrior for illnesses and other prosaic childhood problems. Who will raise these children? Who will be legally responsible for their wellbeing?

Shoigu may envision a future race of extremely capable fighters, but ... thats at least 20 years away, with an added coin flip on nature versus nurture. After all, the Scythian warriors didnt have plumbing, let alone smartphones. This is a whole new world.

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Russia Is Going to Try to Clone an Army of 3,000-Year-Old Scythian Warriors - Popular Mechanics

CAMBRIDGE, Mass. and ZUG, Switzerland and BOSTON, May 12, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced two abstracts detailing updated data from the ongoing CTX001 clinical trials have been accepted for presentation during the European Hematology Association (EHA) 2021 Virtual Congress.

Abstract #EP736 entitled CTX001 for Sickle Cell Disease: Safety and Efficacy Results from the Ongoing CLIMB SCD-121 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells, will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with severe sickle cell disease with more than 3 months of follow-up, as of the interim data cut on January 28, 2021. Data will be updated and information on additional patients will be included for the congress.

Abstract #EP733 entitled CTX001 for Transfusion-Dependent -Thalassemia: Safety and Efficacy Results from the Ongoing CLIMB Thal-111 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells, will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with transfusion-dependent beta thalassemia (TDT) with more than 3 months of follow-up, including patients with the most severe genotypes, as of the interim data cut on January 21, 2021. Data will be updated and information on additional patients will be included for the congress.

The accepted abstracts are now available online on the EHA website https://library.ehaweb.org/eha/#!*menu=6*browseby=8*sortby=2*media=3*ce_id=2035*label=21989*ot_id=25562*marker=1286.

CTX001 is being investigated in two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the Vertex-CRISPR CollaborationVertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics. This amendment is subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, our plans and expectations to present clinical data from the ongoing CTX001 clinical trials during the EHA Virtual Congress, expectations regarding the abstracts that will be made available on the virtual platform, the expectation that data will be updated for the conference, the potential benefits of CTX001, our plans and expectations for our clinical trials and pipeline products, the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and patient enrollment, and our expectations regarding the transaction contemplated by the amended collaboration agreement with CRISPR, including satisfaction of closing conditions and antitrust clearances, and the future activities of the parties pursuant to the amended collaboration agreement. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety and/or efficacy, or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials and clinical trial sites and the clinical trials and clinical trial sites of our collaborators, including patient enrollment, or other reasons, and other risks listed under the heading Risk Factors in Vertex's most recent annual report filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics various clinical programs, including CTX001, including expectations regarding the abstracts that will be made available on the virtual platform and the clinical data that are being presented from the ongoing CTX001 clinical trials during the EHA Virtual Congress; (ii) the timing of the potential closing of the transaction contemplated by the amended collaboration agreement, future activities of the parties pursuant to the collaboration and the potential benefits of CRISPR Therapeutics collaboration withVertex; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain, are neither promises nor guarantees and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final or future trial results; the potential that CTX001 clinical trial results may not be favorable or may not support registration or further development; that future competitive or other market factors may adversely affect the commercial potential for CTX001; the transaction contemplated by the amended collaboration agreement is subject to certain closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act; CRISPR Therapeutics may not realize the potential benefits of the collaboration with Vertex; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; the potential that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orBrenda Eustace, +1 617-341-6187OrManisha Pai, +1 617-429-6891

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

CRISPR Therapeutics Investors:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

Media:Rachel Eides, +1-617-315-4493Rachel.Eides@crisprtx.com

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Vertex and CRISPR Therapeutics to Present New Clinical Data - GlobeNewswire