Industry News & Announcements

Industry News & Announcements

Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS).

Harmony Biosciences

Otsuka Pharmaceutical Development & Commercialization, Inc., (OPDC); Otsuka Pharmaceutical, Co. Ltd. (Otsuka); and Visterra, Inc., an Otsuka group company announce that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigational drug sibeprenlimab for the treatment of immunoglobulin A nephropathy (IgAN; Berger’s disease).

Otsuka America Pharmaceutical, Inc.

The Ministry of Health, Labor and Welfare (MHLW) in Japan has granted marketing and manufacturing authorization for Dupixent® (dupilumab) for the treatment of chronic spontaneous urticaria (CSU) in people aged 12 years and older whose disease is not adequately controlled with existing therapy. Japan is the first country to approve Dupixent for CSU, emphasizing the value of Dupixent as a novel treatment option to manage this disease in patients with unmet needs.

Sanofi

Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") and Kelonia Therapeutics (CEO, Founder: Kevin Friedman, Ph.D., "Kelonia") today announced that Xyphos Biosciences, Inc., (a wholly owned subsidiary of Astellas, "Xyphos") and Kelonia have entered into a research collaboration and license agreement to develop novel Immuno-Oncology therapeutics.

Astellas US LLC

BRENZAVVY Is Now Available to Healthcare Businesses via Cost Plus Drugs Marketplace at business.costplusdrugs.com

TheracosBio today announced that it is extending its collaboration with the Mark Cuban Cost Plus Drug Company. Under the partnership, BRENZAVVY® (bexagliflozin) is available directly to healthcare businesses under the Cost Plus Drugs Marketplace program.

TheracosBio, LLC

Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for inhaled KB707 for the treatment of patients with solid tumors with pulmonary metastases that are relapsed or refractory to standard of care therapy.

Krystal Biotech, Inc.

- Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia may be eligible for treatment -

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.

Vertex Pharmaceuticals

Smith+Nephew (LSE:SN, NYSE:SNN), the global medical technology company, today announces full commercial availability of its AETOS Shoulder System in the US, along with 510(k) clearance for its use with ATLASPLAN◊ 3D Planning Software and Patient Specific Instrumentation for total shoulder arthroplasty. Total shoulder arthroplasty is one of the fastest growing segments in Orthopaedics with an estimated 250,000 procedures in the US by 2025.

Smith & Nephew

Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has authorized SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. SKYCLARYS is the first treatment approved within the European Union for this rare, genetic, progressive neurodegenerative disease.

Biogen

Abbott and Fujirebio announced today a partnership to develop a research use only (RUO) neurofilament-light chain (Nf-L) neurology biomarker assay for use on Abbott’s Alinity® i. Abbott will make the RUO assay available to researchers conducting studies demonstrating the utility of the Nf-L biomarker.

Abbott Laboratories

Abbott and Fujirebio announced today a partnership to develop a research use only (RUO) neurofilament-light chain (Nf-L) neurology biomarker assay for use on Abbott’s Alinity® i. Abbott will make the RUO assay available to researchers conducting studies demonstrating the utility of the Nf-L biomarker.

Fujirebio Diagnostics, Inc.

Tyber Medical LLC, a leading orthopedic device manufacturer providing private label orthopedic implants for the trauma, extremity, and spine markets, has received a U.S. Food and Drug Administration (FDA) 510(k) approval for its Proximal Tibia Plating System. The Tyber Medical System consists of two indication categories: Complete Articular and Partial Articular Fractures.

Tyber Medical, LLC

Gilead Sciences, Inc. and CymaBay Therapeutics, Inc. (Nasdaq: CBAY) announced today a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion. The addition of CymaBay’s investigational lead product candidate, seladelpar for the treatment of primary biliary cholangitis (PBC) including pruritus, complements Gilead’s existing liver portfolio and aligns with its long-standing commitment to bringing transformational medicines to patients.

Gilead Sciences

akeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) has approved EOHILIA (budesonide oral suspension), the first and only FDA-approved oral therapy for people 11 years and older with eosinophilic esophagitis (EoE).1 It will be available in 2 mg/10 mL convenient, single-dose stick packs by the end of February.

Takeda Pharmaceuticals USA, Inc.

AbbVie (NYSE: ABBV) announced today that it has completed its acquisition of ImmunoGen (NASDAQ: IMGN). With the completion of the acquisition, ImmunoGen is now part of AbbVie.

AbbVie, Inc.

Breakthrough Therapy Designation for nipocalimab based on results from the Phase 2 UNITY clinical trial for HDFN

Johnson & Johnson today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for nipocalimab for the treatment of alloimmunizeda pregnant individuals at high risk of severe hemolytic disease of the fetus and newborn (HDFN).

Johnson & Johnson Innovation

Johnson & Johnson today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for nipocalimab for the treatment of alloimmunizeda pregnant individuals at high risk of severe hemolytic disease of the fetus and newborn (HDFN).

Johnson & Johnson

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Redx Pharma plc (AIM: REDX) today announced that the companies have signed a definitive agreement under which Jazz will acquire Redx's KRAS (Kirsten rat sarcoma virus) inhibitor program. Jazz and Redx will collaborate to advance candidates through IND-enabling studies; Jazz will be responsible for all clinical development, regulatory, manufacturing and commercialization activities.

Jazz Pharmaceuticals plc

With an upfront payment of $100 million, Kyowa Kirin has bought into a partnership with BridgeBio to develop and commercialize Truseltiq (infigratinib) in Japan. Besides the sizable upfront installment, Kyowa Kirin agreed to fork over undisclosed payments if the drug reaches certain milestones, plus pay royalties up to the “high-twenties" in percentage terms.

Kyowa Kirin USA Holdings, Inc.

Cencora, Inc. (NYSE: COR) today announced the closing of its public offering of $500 million aggregate principal amount of its 5.125% Senior Notes due 2034 (the “Notes”), in an underwritten registered public offering. The offering was made pursuant to an effective shelf registration statement Cencora filed with the Securities and Exchange Commission (the “SEC”) on November 23, 2021.

Cencora (formerly AmerisourceBergen)

The ALS Association Greater Philadelphia Chapter has completed rebranding to become ALS United Mid-Atlantic. This transition comes after the Greater Philadelphia Chapter joined 14 other Chapters in legally separating from The ALS Association on September 1, 2023. These chapters represent approximately half of the ALS patient population and Certified Treatment Centers and ALS Clinics in the nation.

ALS Association Greater Philadelphia Chapter

Helius Medical Technologies, Inc. (Nasdaq:HSDT) (“Helius” or the “Company”), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait deficits, announced today that it has concluded its interaction with the U.S. Food and Drug Administration (“FDA”) on optimizing the development plan for its stroke program.

Helius Medical Technologies

Tyber Medical LLC, a leading orthopedic device manufacturer providing private label orthopedic implants for the trauma, extremity, and spine markets, received clearance for the anatomical plating system in Canada. The comprehensive portfolio previously received FDA 510(k) in the US and has now been cleared through Health Canada.

Tyber Medical, LLC

NRx Pharmaceuticals, Inc. (Nasdaq: NRXP) ("NRx Pharmaceuticals", the "Company"), a clinical-stage biopharmaceutical company, today announced the incorporation of HOPE Therapeutics™, a biotechnology company dedicated to bringing NRX-100 (IV Ketamine), which will be re-designated HTX-100, a potentially lifesaving treatment option for patients with Suicidal Depression.

NRx Pharmaceuticals, Inc.

Novo Holdings, the investment arm of Novo Nordisk’s parent foundation, is buying Catalent in a deal valued at $16.5 billion that’s the biggest it’s ever made.

Novo Nordisk

Merck MRK 0.55%increase; green up pointing triangl& Co. has struck a deal to buy Elanco ELAN 0.75%increase; green up pointing triangAnimal Health’s aqua business in a deal that expands the drugmaker’s animal-health business.

Merck & Co., Inc.

A Main Line pharmaceutical developer is creating a new company to bring its experimental drug candidate targeting suicidal depression to patients, should that medicine receive regulatory approval. NRx Pharmaceuticals of Radnor said the new business, called Hope Therapeutics, will initially be owned by NRx and its current shareholders via a planned tax-free dividend with an accompanying royalty coupon tied to potential future sales of the drug.

NRx Pharmaceuticals, Inc.

In a move designed to advance opportunities for students to expand their access to cutting-edge research and education in the field of biomedical sciences, Philadelphia College of Osteopathic Medicine (PCOM) and The Wistar Institute have announced an academic collaboration to offer degree programs, courses and other educational opportunities to students at each institution.

The Wistar Institute

Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201.

Cabaletta Bio, Inc.

KaliVir Immunotherapeutics, Inc., a biotech company developing cutting-edge, multi-mechanistic oncolytic viral immunotherapy programs, has unveiled its state-of-the-art GMP cleanroom facility to manufacture its pipeline of oncolytic viruses. KaliVir has already successfully executed the inaugural manufacturing run for its lead clinical candidate, VET3-TGI

KaliVir Immunotherapeutics, Inc.

The William G. Rohrer Charitable Foundation has awarded the Coriell Institute for Medical Research $500,000 to develop the Coriell-Cooper Cancer Biobank, a new collection of biological samples derived from people undergoing treatment for cancer. The grant, structured for disbursement over five years, will allow for the acquisition of needed equipment and staff to support the creation and growth of this important collection.

Coriell Institute for Medical Research

If approved, afami-cel will be the first engineered T-cell therapy for solid tumors and the first effective treatment option for synovial sarcoma in more than a decade

Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review its Biologics License Application (BLA) for afami-cel, an investigational engineered T-cell therapy for advanced synovial sarcoma. The application has a Prescription Drug User Fee Act (PDUFA) target action date of August 4, 2024.

Adaptimmune LLC

Study to assess safety and immunogenicity of novel protein nanoparticle vaccine candidates UVAX-1107 and UVAX-1197 in healthy volunteers

Uvax Bio, LLC, (“Uvax” or “the Company”) a privately held, clinical-stage vaccine company utilizing rational and computational biology to design and deliver novel 1c-SApNP® protein-based vaccines, today announced the first participant has been dosed in a Phase 1 clinical trial evaluating the Company’s HIV-1 vaccine candidates, UVAX-1107 and UVAX-1197, in healthy volunteers in Australia.

Uvax Bio, LLC

Tobin Scientific proudly announces the attainment of ISO 9001 certification, a significant step in its ongoing commitment to quality and operational excellence. This achievement reflects the stringent quality standards consistently upheld across Tobin Scientific's expanding cGMP facilities. As the first certification of its kind within their network, it establishes a benchmark for the level of quality and excellence that Tobin Scientific is committed to in its comprehensive cGMP operations.

Tobin Scientific

Through a pilot program aimed at accelerating and supporting life sciences startups in our state, the Pennsylvania Biotechnology Center (PABC) will provide financial support and free lab space to more than a dozen startups at the PABC in Doylestown, its B+labs incubator in Philadelphia and elsewhere in Pennsylvania.

Pennsylvania Biotechnology Center

Tyber Medical Broadens Its Vast Plating Portfolio with FDA Approval of Additional Screw and Plate Options, and Indications for Mini-Frag System

Tyber Medical, LLC, a leading orthopedic device manufacturer providing private label orthopedic implants for the trauma, extremity and spine markets, has received a U.S. Food and Drug Administration (FDA) 510(k) approval for a line extension to the existing, approved Mini-Frag System, with additional screw and plate options, and indications. This additional clearance expands Tyber Medical products' healing capabilities and adds to their restorative portfolio.

Tyber Medical, LLC

Takeda today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID® [Immune Globulin Infusion (Human) 10% solution] as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP).

Takeda Pharmaceuticals USA, Inc.

At Sanofi and Regeneron, it’s all about the kids—at least with respect to their recent advancements of anti-inflammatory powerhouse Dupixent. On Thursday, the FDA signed off on the IL-4 receptor alpha antagonist to treat children between the ages of 1 and 11 who weigh at least 33 pounds with eosinophilic esophagitis (EoE). Dupixent becomes the first treatment in the U.S. for children in this age group with the disorder.

Sanofi

At Sanofi and Regeneron, it’s all about the kids—at least with respect to their recent advancements of anti-inflammatory powerhouse Dupixent. On Thursday, the FDA signed off on the IL-4 receptor alpha antagonist to treat children between the ages of 1 and 11 who weigh at least 33 pounds with eosinophilic esophagitis (EoE). Dupixent becomes the first treatment in the U.S. for children in this age group with the disorder.

Regeneron Pharmaceuticals, Inc.

The $83.9 million gift from P. Roy and Diana Vagelos, announced Thursday by Penn, follows earlier donations from the couple, who previously funded the creation of an energy science center.

The University of Pennsylvania’s School of Arts & Sciences has received a nearly $84 million gift for science initiatives — the largest in the school’s history. Most of the $83.9 million gift from P. Roy and Diana Vagelos, announced Thursday by Penn, will go toward graduate education in the Department of Chemistry, including the establishment of 20 new Vagelos fellows, according to the university.

Penn Medicine

EpiVario Inc., a biotech company pioneering neuroepigenetics to treat addiction disorders, today announced that it has entered into an asset purchase agreement with Metabomed LTD ("Metabomed") to acquire Metabomed's small molecule acetyl co-A synthetase-2 inhibitor ("ACSS2i") portfolio.

EpiVario Inc.

Neuraptive Therapeutics Inc., a leader in innovative medical solutions, is pleased to announce that it has received Orphan Drug Designation from the United States (US) Food and Drug Administration (FDA) for its fusogen contained in the NTX-001 treatment kit. This designation marks a significant milestone in developing NTX-001, a novel treatment for peripheral nerve injury requiring repair.

Neuraptive Therapeutics, Inc.

WuXi Biologics ("WuXi Bio") (2269. HK), a leading global Contract Research, Development, and Manufacturing Organization (CRDMO), today announced that its Research Service Unit (R in CRDMO) has signed a research service agreement with BioNTech SE (Nasdaq: BNTX, "BioNTech"), a next generation immunotherapy company pioneering novel therapies for cancer and other serious diseases.

WuXi Biologics

GSK will pay $1 billion upfront to buy clinical-stage asthma drugmaker Aiolos Bio, the British drugmaker said early Tuesday morning. It could pay up to $400 million more if milestones are met.

GSK

Venatorx Pharmaceuticals and Menarini Group announced that they have entered into an agreement under which Menarini will acquire the exclusive rights to commercialize, upon approval of relevant health authorities, cefepime-taniborbactam in 96 countries in Europe, Latin America, Middle East, Turkey and North Africa and the Commonwealth of Independent States (CIS).

Venatorx Pharmaceuticals, Inc.

Johnson & Johnson is adding to Big Pharma’s love for antibody drug conjugates with a $2 billion cash deal to swoop up Ambrx Biopharma.

Johnson & Johnson Innovation

– Second and third FDA Fast Track Designations for CABA-201, following the systemic lupus erythematosus (SLE) and lupus nephritis (LN) designation, providing the opportunity for expedited development and review of CABA-201 for the treatment of these autoimmune diseases –

Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted separate Fast Track Designations to CABA-201.

Cabaletta Bio, Inc.

NeuMoDx CT/NG Assay designed to improve accessibility, affordability and timeliness in sexually transmitted infection (STI) testing in the U.S. // U.S. regulatory clearance of new assay for use on both NeuMoDx 96 and 288 versions of integrated PCR-based clinical molecular testing system // Global presence anchored by 16 EU-certified in-vitro diagnostics (CE-IVD) tests, positioning NeuMoDx as having one of the broadest test menus available to customers

QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced the U.S. Food and Drug Administration (FDA) clearance for the NeuMoDx CT/NG Assay 2.0, growing its test menu for its integrated PCR-based clinical molecular testing systems NeuMoDx 96 and 288 in the United States.

QIAGEN Sciences Inc

Merck & Co. has struck a deal to buy clinical-stage immunotherapy company Harpoon Therapeutics for about $680 million.

Merck & Co., Inc.

Neuraptive Therapeutics, Inc., a leader in nerve repair and regeneration, is pleased to announce a significant milestone in transforming the treatment paradigm for peripheral nerve injuries. The successful demonstration of proof of concept in the NEUROFUSE study of NTX-001 marks an unprecedented advancement in providing hope for faster and more complete functional recovery to individuals impacted by peripheral nerve transections.

Neuraptive Therapeutics, Inc.

Herman O. West founded the drug packaging manufacturer in Fishtown in 1923. From packaging penicillin during World War II to going public in 1970, here's why the Philly region remains home.

Philly has been getting a lot of attention lately for its life sciences success, with this past October’s EDA Tech Hubs designation for precision medicine and a swell of collaborative support from regional stakeholders. But its history goes back further than you might expect.

West Pharmaceutical Services, Inc.

After more than 20 years, the foundation ownership working together as Almono LLC has Pittsburgh's two major universities moving forward on what are expected to be catalytic development projects for brownfield redevelopment in Hazelwood.

Carnegie Mellon University

Starting next summer West Chester University will have a new president. The Board of Governors of Pennsylvania’s State System of Higher Education recently announced that it has selected Dr. R. Lorraine Bernotsky to be the next president of West Chester University (WCU). The appointment takes effect July 1, 2024.

West Chester University

B. Braun Medical Inc. of Bethlehem recently announced the launch of its new CARESITE Micro Luer Access Device, designed to reduce exposure to harmful chemicals and decrease infection risk, making IV access safer for patients and healthcare providers.

B. Braun Medical Inc.

Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, today announced that it entered into a definitive asset purchase agreement with Atreca, Inc. (Nasdaq: BCEL) to acquire a collection of antibody-related assets and materials.

Immunome, Inc.

A $6 million grant awarded to Lehigh University is expected to help solve a common problem within universities: getting the scientific discoveries of its faculty, graduate students and postdoctoral researches out of the research stage.

Lehigh University

Partners with Cook MyoSite to establish a state-of-the-art facility to conduct human islet isolations for hospital systems without in-house capabilities

Imagine Pharma, a biotech company focused on advancing the commercialization of its novel IMG-1 polypeptide in Oral Delivery, Therapeutics, and Regenerative Medicine, announced today that it has successfully completed more than 32 clinical islet cell isolation procedures for several leading hospital systems across the United States, including the Cincinnati Children's Hospital Medical Center.

Imagine Pharma LLC

Gilead Sciences is uploading $60 million to Compugen’s bank account plus hefty milestones down the road in exchange for the rights to a preclinical IL-18 binding protein program that includes a solid tumor asset.

Gilead Sciences

Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to beremagene geperpavec-svdt (B-VEC or VYJUVEK) for the treatment of dystrophic epidermolysis bullosa (DEB).

Krystal Biotech, Inc.

On December 7, 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation ("ODD") to NXL-004, an investigational AAV gene therapy product being developed by NeuExcell Therapeutics Inc. ("NeuExcell"), for the treatment of malignant glioma. This is the world's first AAV gene therapy product for malignant glioma to receive FDA ODD designation.

NeuExcell Therapeutics inc.

Tyber Medical, LLC, a leading orthopedic device manufacturer providing private label orthopedic implants for the trauma, extremity, and spine markets, received a U.S. Food and Drug Administration (FDA) 510(k) clearance for its new Distal Radius Plating System.

Tyber Medical, LLC

Leave it to Carnegie Mellon University to include a robot in the ceremonial shoveling to commemorate the construction start of its new $100 million development investment at Hazelwood Green.

Carnegie Mellon University

Pfizer’s $43 billion acquisition of Seagen is in the books, the company said on Thursday, as it becomes the largest M&A transaction in the biopharma industry since AbbVie snatched up Allergan for $63 billion in 2019.

Pfizer Inc.

First and only hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor approved for these adult patients with advanced RCC

Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved WELIREG, an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, for the treatment of adult patients with advanced renal cell carcinoma (RCC) following a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

Merck & Co., Inc.

ECUR-506 is a gene editing-based investigational therapy for the treatment of Ornithine Transcarbamylase (OTC) deficiency

iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today that the Australian Therapeutic Goods Administration (TGA) has approved the Company’s Clinical Trial Notification (CTN) for ECUR-506 (formerly GTP-506), an investigational therapy in development for the treatment of Ornithine Transcarbamylase (OTC) deficiency in pediatric (or neonatal) patients.

iECURE, Inc.

Prelude Therapeutics Incorporated (Nasdaq: PRLD) (“Prelude” or the “Company”), a clinical-stage precision oncology company, today announced a private placement that the Company estimates will result in gross proceeds of approximately $25 million before deducting estimated offering expenses payable by the Company. Proceeds from the private placement will be used to primarily fund the continued advancement of its SMARCA2 portfolio, for working capital and general corporate purposes.

Prelude Therapeutics

The CRISPR treatment enables people with sickle cell disease to make a normal form of hemoglobin. Other such treatments are being tested for cancer and heart disease.

CHOP

Actuated Medical, a leading innovator in medical device technologies, announced today the successful acquisition of Mitotyping Technologies LLC, a prominent DNA forensic company specializing in advanced mitochondrial DNA analysis. This strategic move reflects Actuated Medical’s commitment to diversification and growth, expanding its capabilities into the field of forensic science.

Actuated Medical, Inc.

Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drugs for immuno-inflammatory diseases, today announced an exclusive patent license agreement with Sun Pharmaceutical Industries, Inc. (Sun Pharma).

Aclaris Therapeutics, Inc.

Century Therapeutics (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology and autoimmune and inflammatory disease, today announced that the Company has been notified by the U.S. Food and Drug Administration (FDA) that the Company’s Phase 1 clinical trial may proceed to assess CNTY-101 in patients with moderate to severe systemic lupus erythematosus (SLE) who have failed at least two standard immunosuppressive therapies.

Century Therapeutics

Novartis today announced that the U.S. Food and Drug Administration (FDA) approved Fabhalta® (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)1.

Novartis

Amicus Therapeutics (Nasdaq: FOLD) today announced that John F. Crowley, Executive Chairman of Amicus Therapeutics, will step down from the Amicus Board of Directors and assume the role of President and Chief Executive Officer of BIO, the Biotechnology Innovation Organization, effective March 4, 2024.

Amicus Therapeutics

West Pharmaceutical Services, Inc. (NYSE: WST), a global leader in innovative solutions for injectable drug administration systems, today announced FDA 510(k) clearance and launch of its Vial2Bag Advanced® 13mm admixture device.

West Pharmaceutical Services, Inc.

ohnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted TAR-200 Breakthrough Therapy Designation (BTD) for the potential future treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC), who are ineligible for or elected not to undergo radical cystectomy (surgical removal of the bladder).

Johnson & Johnson Innovation

CT-0525 is the first CAR-Monocyte to be evaluated in humans in the solid tumor setting First patient expected to be treated in the first half of 2024

Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced the clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA) for CT-0525, an ex vivo gene-modified autologous chimeric antigen receptor-monocyte (CAR-Monocyte) cellular therapy intended to treat solid tumors that overexpress human epidermal growth factor receptor 2 (HER2).

Carisma Therapeutics, Inc.

Funds will support progression of Aro’s pipeline of Centyrin-siRNA programs, including ABX1100, a novel potential treatment for Pompe Disease.

PHILADELPHIA--(BUSINESS WIRE)--Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced it has secured $41.5M in a Series B funding round.

Aro Biotherapeutics Company

Tyber Medical has been selected to receive the Governor's Award for Safety Excellence (GASE)—a highly competitive program that acknowledges exceptional safety and prevention initiatives in the workplace as well as the effective and successful management of those initiatives by its employees.

Tyber Medical, LLC

A Philadelphia biotechnology company working to discover and develop more effective drugs by testing them on lab-grown human organs said Wednesday it has raised $38 million in a private stock sale.

Vivodyne, Inc.

Acquisition underscores Merck’s ongoing commitment to developing treatments for neurodegenerative diseases

RAHWAY, N.J., & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside of the United States and Canada, and Caraway Therapeutics, Inc. announced today that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire Caraway Therapeutics for a total potential consideration of up to $610 million, including an undisclosed upfront payment as well as contingent milestone payments.

Merck & Co., Inc.

BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Nov. 16, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Vertex Pharmaceuticals

After more than two years of testing, a Philadelphia medical technology company has officially launched an artificial intelligence-based operating system it created to dramatically accelerate the identification and development of promising drug candidates.

BioPhy

PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) this week announced that the Company’s fourth Investigational New Drug (IND) application for CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, has been allowed to proceed by the U.S. Food and Drug Administration (FDA) for a Phase 1/2 study in patients with generalized myasthenia gravis (gMG).

Cabaletta Bio, Inc.

Melinta Therapeutics LLC (“Melinta”) and Venatorx Pharmaceuticals, Inc. (“Venatorx”), today announced that they have entered into a License Agreement to facilitate a strategic partnership in the U.S. to commercialize cefepime-taniborbactam, a beta-lactam / beta-lactamase inhibitor (BL/BLI) combination antibiotic being developed for the treatment of complicated urinary tract infections (cUTI) and hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) in adults.

Venatorx Pharmaceuticals, Inc.

A biotech company working to commercialize products relating to regenerative medicine, oral delivery and therapeutics has closed on a $32.5 million investment round to help advance that work.

Imagine Pharma LLC

The U.S. Food and Drug Administration approved Wezlana (ustekinumab-auub) as a biosimilar to and interchangeable with Stelara (ustekinumab) for multiple inflammatory diseases.

Life Sciences Pennsylvania

Sanofi has announced the winners of its 2023 Health Equity Accelerator Awards, honoring 10 nonprofit organizations that exemplify the company's ambition to provide equitable access to healthcare using innovative solutions for hard-to-solve health disparities.

Sanofi

Ben Franklin Technology Partners of Northeastern Pennsylvania said it has approved $2.3 million in investments for the first quarter of Fiscal Year 2024. A total of 10 innovative companies, including eight startups and two manufacturers, received funding.

Ben Franklin Technology Partners, SE PA

This week's lab notes has items on two venture fund closings, an honor for the former leader of the Biotechnology Innovation Organization, the completion of a South Jersey biotech company's asset sale and more.

1315 Capital, LLC

Integral Molecular, a leading provider of reagents for vaccine evaluation, announces the launch of TiterSafe™ influenza virus-like particles, an off-the-shelf virus-alternative for key experiments in testing influenza vaccines.

Integral Molecular

NRx Pharmaceuticals, Inc. (Nasdaq: NRXP) ("NRx Pharmaceuticals", (NRx), a clinical-stage biopharmaceutical company and Nephron Pharmaceuticals, Inc. (Nephron), a leading manufacturer of sterile injectable drugs today announced the signing of a development and manufacturing agreement to manufacture a presentation of ketamine suitable for treating suicidal depression

NRx Pharmaceuticals, Inc.

NEW YORK & MAINZ, Germany--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced that the U.S. Food and Drug Administration (FDA) approved the supplemental Biologics License Application (COMIRNATY 2023-2024 Formulation) for individuals 12 years and older and granted emergency use authorization for individuals 6 months through 11 years of age for the companies’ Omicron XBB.1.5-adapted monovalent COVID-19 vaccine.

Pfizer Inc.

PHILADELPHIA, Sept. 7, 2023 /PRNewswire/ -- BlueWhale Bio, Inc., a new company spun out of the University of Pennsylvania to overcome key bottlenecks in cell and gene therapy manufacturing, announced today that it has raised $18 million in Seed financing. The round was led by Danaher Ventures LLC, the corporate venture subsidiary of Danaher Corporation, with a deep syndicate of leading life science investors, including Novalis LifeSciences and Marshall Wace, amongst others.

James Riley Ph.D.

Verismo Therapeutics, a clinical-stage CAR-T company developing novel KIR-CAR platform technology invented at and licensed from the University of Pennsylvania today announced that a first patient has been dosed in their STAR-101 clinical trial to study the SynKIR-110 product in individuals with advanced ovarian cancer, mesothelioma, and cholangiocarcinoma.

Verismo Therapeutics

Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).

Krystal Biotech, Inc.

Emergent has officially shipped its over the counter, original prescription strength NARCAN® Naloxone HCl Nasal Spray 4 mg to leading mass, drug/pharmacy and grocery stores, as well as online retailers NARCAN® Nasal Spray will be the first, life-saving emergency medicine for opioid overdose available on shelves beginning this September with a Manufacturer’s Suggested Retail Price of $44.99

Emergent BioSolutions

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that an individual in Germany with severe hemophilia A was treated with ROCTAVIAN® (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.

BioMarin Pharmaceutical, Inc.

Acquisition expands pipeline and diversifies portfolio to drive long-term growth Innovative potential new therapeutic option for rare/orphan neuropsychiatric disorders with high unmet medical needs Lead asset in pivotal Phase 3 trial for Fragile X syndrome and has completed Phase 2 proof-of-concept study in 22q11.2 deletion syndrome

Harmony Biosciences

HORSHAM, Pa., Aug. 11, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the U.S. Food & Drug Administration (FDA) has approved AKEEGA™ (niraparib and abiraterone acetate), the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive mCRPC, as detected by an FDA-approved test.1

Johnson & Johnson Innovative Medicine (formerly Janssen)

HORSHAM, Pa., Aug. 10, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of TALVEY™ (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Johnson & Johnson Innovative Medicine (formerly Janssen)

The FDA has signed off on Iveric’s geographic atrophy (GA) treatment Izervay. The nod comes less than three weeks after Astellas’ lone rival in the indication, Apellis’ newly approved Syfovre, was flagged for severe side effects.

Astellas US LLC

PRINCETON, N.J., August 2, 2023 – Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. announced today that the U.S. Food and Drug Administration (FDA) has approved LONSURF® (trifluridine/tipiracil) as a single agent or in combination with bevacizumab for the treatment of adult patients with metastatic colorectal cancer (mCRC) previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type, an anti-EGFR therapy.

Taiho Oncology, Inc.

A cell therapy developer spun out of the University of Pennsylvania three years ago has raised $17 million to advance its lead immunotherapy candidates targeting a variety of cancers.

Verismo Therapeutics

Today, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

BioMarin Pharmaceutical, Inc.

Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) has approved NGENLA (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. NGENLA is expected to become available for U.S. prescribing in August 2023.

Pfizer Inc.

Annovis Bio, Inc. (NYSE: ANVS), a clinical stage drug platform company developing novel and transformative therapies for neurodegenerative diseases, announces the creation of a novel crystalline form of buntanetap (ANVS402), and the filing of a new composition of matter provisional patent with the U.S. Patent and Trademark Office.

Annovis Bio, Inc.

Brixadi was approved by the FDA as an extended-release, injectable medication available in weekly and monthly doses.

Braeburn

Amgen announced that the U.S. Food and Drug Administration has approved the supplemental Biologics License Application (sBLA) for BLINCYTO® (blinatumomab) for the treatment of adults and pediatric patients with CD19-positive B-cell precursor acute lymphoblastic leukemia in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%, based on additional data from two Phase 3 studies that were submitted. The approval converts BLINCYTO's accelerated approval to a full approval.

Amgen, Inc.

HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B, has been proven to elevate and sustain factor IX levels for years, significantly reduce the rate of annual bleeds versus standard of care, and reduce or eliminate the need for prophylactic treatment in 94% of patients (51 out of 54) after a one-time infusion

CSL Behring

The BIO International Convention was last held in Philadelphia in 2019 and generated an economic impact of $41 million for the region. The annual event attracted 20,000 attendees last week in Boston.

Biotechnology Innovation Organization

Lynparza can now be used in combination with Johnson & Johnson’s Zytiga and a corticosteroid to treat patients with mCRPC whose tumors have BRCA mutations.

Merck & Co., Inc.

Krystal Biotech Inc. (NASDAQ: KRYS) announced that the U.S. Food and Drug Administration approved the use of VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) for patients who are six months of age or older.

Krystal Biotech, Inc.

Optinose (NASDAQ:OPTN), a pharmaceutical company focused on patients treated by ear, nose and throat (ENT) and allergy specialists, today announced the U.S. Food and Drug Administration (FDA) accepted for review its supplemental new drug application (sNDA) requesting approval of XHANCE as a treatment for chronic rhinosinusitis.

Optinose

PHILADELPHIA–Integral Molecular, a founding member of Philadelphia’s biotech community, announces the official opening of its 32,000 square foot headquarters and integrated research facilities that will include its pandemic preparedness laboratories.

Integral Molecular

Otsuka America Pharmaceutical, Inc. (Otsuka) and Lundbeck announce the U.S. Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for ABILIFY ASIMTUFII® (aripiprazole) extended-release injectable suspension for intramuscular use, a once-every-two-months injection for the treatment of schizophrenia in adults or for maintenance monotherapy treatment of bipolar I disorder in adults.

Otsuka America Pharmaceutical, Inc.

Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved PREVNAR 20® (20-valent Pneumococcal Conjugate Vaccine) for the prevention of invasive pneumococcal disease (IPD) caused by the 20 Streptococcus pneumoniae (pneumococcal) serotypes contained in the vaccine in infants and children six weeks through 17 years of age, and for the prevention of otitis media in infants six weeks through five years of age caused by the original seven serotypes contained in PREVNAR®.

Pfizer Inc.

The Food and Drug Administration granted marketing approval Wednesday to Zynyz, a drug developed by Wilmington-based Incyte to treat advanced Merkel cell carcinoma.

Incyte Corporation

Silicon Valley Bank's failure — the largest in the U.S. since the 2008 financial crisis — has spawned shockwaves across the U.S. startup scene and sent some companies scrambling to secure funds for payroll and other expenses.

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