On May 7, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company will present at the following conferences (Press release, BioCryst Pharmaceuticals, MAY 7, 2024, View Source [SID1234642763]):

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Citizens JMP Life Sciences Conference in New York on Monday, May 13, 2024, at 12:30 p.m. ET.
2024 RBC Capital Markets Global Healthcare Conference in New York on Tuesday, May 14, 2024, at 3:05 p.m. ET.
Bank of America Health Care Conference 2024 in Las Vegas on Wednesday, May 15, 2024, at 5:20 p.m. ET.
H.C. Wainwright 2nd Annual BioConnect Investor Conference in New York on Monday, May 20, 2024, at 10:30 a.m. ET.
Jefferies Global Healthcare Conference in New York on Thursday, June 6, 2024, at 9:30 a.m. ET.
Links to the live audio webcasts and replays of the presentations may be accessed in the Investors & Media section of BioCryst’s website at http://www.biocryst.com.

On May 7, 2024 Bio-Techne Corporation reported it will showcase its portfolio of cell and gene therapy workflow solutions at the upcoming annual meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place May 7-11, 2024, in Baltimore, Maryland (Press release, Bio-Techne, MAY 7, 2024, View Source [SID1234642762]).

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Bio-Techne’s booth (#1627) will feature our market-leading portfolio of high-quality life science research use only (RUO) and Good Manufacturing Practice (GMP) reagents, immunoassays, automated proteomic analytical instruments, and multi-omics solutions for accelerating cell and gene therapy development and manufacturing. We will also feature our "Immune Cell Therapy Workflow" multimedia experience, which enables attendees to explore how Bio-Techne’s solutions streamline and accelerate the immune cell processing workflow.

Bio-Techne will host three speaker sessions during the conference, featuring both internal experts and customer case studies. The topics covered will include characterizing biodistribution and safety of cell and gene therapies, protein analytics strategies for gene therapy, and advancements in closed systems and non-viral cell engineering for immune cell therapy manufacturing.

Additionally, several Bio-Techne scientists will present posters highlighting our in-house studies in the areas of immune cell culture, automated spatial multi-omics, and protein or viral vector characterization. These include investigations into the efficient activation and expansion of human T cells for CAR-T therapy, RNA/protein ISH imaging of gene therapy biodistribution and persistence, and the multiparameter characterization of adeno-associated virus capsids.

"We are proud to support the advancement of cell and gene therapy research, development and manufacturing with our robust portfolio of innovative tools, reagents and workflow solutions," said Kim Kelderman, Bio-Techne’s President and Chief Executive Officer. "Bio-Techne’s portfolio plays a critical role in the development of next-generation cell and gene therapies. Our broad presence at ASGCT (Free ASGCT Whitepaper) will showcase the many roles Bio-Techne plays in catalyzing advances in science and medicine, and ultimately enabling patients to live healthier and longer lives."

Bio-Techne Presentations:
Novel RNAscope Multi-omics Spatial Approach to Characterize Biodistribution and Safety of Cell and Gene Therapies

Wednesday, May 8th from 4:45pm – 5:15pm in Room 337-338
Presented by Sharel Figueredo, Ph.D.
Protein Analytics Strategies for Gene Therapy – from Viral Vector Characterization to Clinical Trials

Thursday, May 9th from 8:30am – 9:30am in Room 324-326
Presented by Hiren Patel, Ph.D. (REGENEXBIO Inc.)
Moderator Chris Heger, Ph.D.
Advancements in GMP and Closed System Reagents for Immune Cell Therapy Manufacturing

Wednesday, May 8th from 3:45pm -4:00pm in the Exhibit Hall
Presented by Miles Smith, Ph.D.
Bio-Techne Poster Presentations:
Efficient T-cell Activation and Expansion for CAR-T Therapy is Dependent on a Complex Interplay of the Starting Donor Population, Activation Antibodies and Expansion Platform

Wednesday, May 8th at 12:00 pm
Presenter: Hatt Heidtman, Ph.D.
Abstract 868
Next Generation Protein Characterization of CD19-CAR Signaling Cascades

Thursday, May 9th at 12:00 pm
Presenter: Charles Haitjema, Ph.D.
Abstract 1329
A Versatile Assay for Same-Section Spatial Visualization of Small RNA, mRNA, Exon Junction, Protein and Protein-Protein Interactions

Thursday, May 9th at 12:00 pm
Presenter: Maithreyan Srinivasan, Ph.D.
Abstract 1397
RNA/Protein Multiomics Imaging Using RNAscope Technologies to Understand Nonclinical Biodistribution (BD) and Persistence of Gene Therapy (GT) Products

Thursday, May 9th at 12:00 pm
Presenter: Maithreyan Srinivasan, Ph.D.
Abstract 1398
Characterization of Adeno-Associated Virus Capsids by a Novel Capillary Isoelectric Focusing Based Fractionation Method and Charge-Detection Mass Spectrometry

Friday, May 10th at 12:00 pm
Presenter: Chris Heger, Ph.D.
Abstract 1463

On May 7, 2024 – Beam Therapeutics Inc.(Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported first quarter 2024 financial results and provided updates across the company’s hematology and genetic disease franchises (Press release, Beam Therapeutics, MAY 7, 2024, View Source [SID1234642761]).

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"We’re pleased to share progress across our high-priority programs that exemplify our commitment to rapid, focused execution and a dedication to developing differentiated, one-time medicines for serious genetic diseases," said John Evans, chief executive officer of Beam. "We have successfully completed dosing and engraftment for three sickle cell disease patients in the sentinel cohort of the BEAM-101 BEACON trial allowing us to now move forward with expansion phase dosing. We look forward to sharing data for multiple patients treated with BEAM-101 later this year. In addition, our team has done an incredible job executing our ex-U.S. clinical strategy for BEAM-302, securing CTA clearance in the UK and rapidly working toward the initiation of our Phase 1/2 trial in patients with alpha-1 antitrypsin deficiency. This study is designed to demonstrate proof-of-concept for correction of the disease-causing mutation that could potentially help patients with both lung and liver disease manifestations. These updates, supported by our robust balance sheet, mark a significant stride toward our goal of establishing base editing as a potentially transformative and differentiated therapeutic option for patients in need."

First Quarter 2024 and Recent Progress

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Sequential dosing and engraftment have been successfully completed for the three patients in the sentinel cohort of the BEACON Phase 1/2 clinical trial of BEAM-101 in severe sickle cell disease.
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Following clearance by the data monitoring committee, the expansion cohort of the BEACON trial of BEAM-101, in which patients can be dosed in parallel, is now open with dosing expected to begin imminently.
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In March 2024, Beam announced the clearance of its clinical trial authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare Products Regulatory Agency for BEAM-302, the company’s priority genetic disease program for the treatment of alpha-1 antitrypsin deficiency (AATD).
Key 2024 Anticipated Milestones

Hematology Franchise

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In the BEACON Phase 1/2 clinical trial of BEAM-101 in adults with severe sickle cell disease, Beam anticipates continuing to enroll and dose patients in the expansion cohort of the trial, with a total target of up to 45 treated patients.
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The company expects to report data from multiple patients in the BEACON trial in the second half of 2024.
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Beam continues to advance and invest in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning platform and anticipates initiating Phase 1-enabling preclinical studies for the program in 2024.
Genetic Disease Franchise

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Beam expects to initiate the Phase 1/2 clinical trial for BEAM-302 in AATD in the first half of 2024.
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Beam expects to submit an investigational new drug (IND) application in the U.S. for BEAM-301 for the potential treatment of glycogen storage disease type 1a (GSD1a) in the first half of 2024.

First Quarter 2024 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities, were $1.1 billion as of March 31, 2024, compared to $1.2 billion as of December 31, 2023.
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Research & Development (R&D) Expenses: R&D expenses were $84.8 million for the first quarter of 2024, compared to $99.6 million for the first quarter of 2023.
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General & Administrative (G&A) Expenses: G&A expenses were $26.7 million for the first quarter of 2024, compared to $23.5 million for the first quarter of 2023.
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Net Loss: Net loss was $98.7 million for the first quarter of 2024, or $1.21 per share, compared to $96.5 million for the first quarter of 2023, or $1.33 per share.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of March 31, 2024, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into 2027. This expectation includes funding directed toward reaching each of the key anticipated milestones for BEAM-101, ESCAPE, BEAM-301 and BEAM-302 described above, as well as continued investments in platform advancements and manufacturing capabilities.

On May 07, 2024 Avid Bioservices, Inc. (NASDAQ: CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that the company will participate at two upcoming investor conferences. Nick Green, president and chief executive officer, will be the featured speaker in a fireside chat at the RBC Capital Markets 2024 Global Healthcare Conference, and will deliver a corporate presentation at the 2024 Bank of America Securities Healthcare Conference (Press release, Avid Bioservices, MAY 7, 2024, View Source [SID1234642760]).

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Details of the company’s participation are as follows:

RBC Capital Markets 2024 Global Healthcare Conference
Conference Date: May 14-15, 2024
Fireside Chat Time/Date: 4:35 – 5:05 p.m. Eastern on Tuesday, May 14, 2024
Location: InterContinental New York Barclay, New York
2024 Bank of America Healthcare Conference
Conference Date: May 14-16, 2024
Presentation Time/Date: 11:55 a.m. – 12:10 p.m. Eastern on Thursday, May 16, 2024
Location: Encore Hotel, Las Vegas, NV

On May 7, 2024 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) (Aurinia or the Company) reported that it will attend the 2024 RBC Capital Markets Global Healthcare Conference in New York from May 14-15, 2024 (Press release, Aurinia Pharmaceuticals, MAY 7, 2024, View Source [SID1234642759]).

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Aurinia management will host one-on-one meetings with investors and will participate in a fireside chat on Wednesday, May 15, from 10:00 to 10:25 AM Eastern Time. A live webcast of the session will be available on the Investor section of Aurinia’s website, which can be found here.