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The Weekly Pill

05/20/24-05/26/24

May 25, 2024
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Hi everyone,

Welcome to all our new subscribers who joined us this week! We're thrilled to have you as part of The Weekly Pill community. Your readership means the world to us. If you could kindly share this newsletter with your friends or coworkers, we would greatly appreciate your support! This resource holds value for a wide range of individuals, including venture capitalists, students, professionals at all levels within pharmaceutical companies, and anyone with a curious mindset eager to learn. Your assistance in this endeavor is greatly appreciated.

I sincerely hope you enjoy reading our newsletter and that it adds value to your understanding of the biotech world. Wishing each of you a blessed week ahead, and I look forward to connecting with you all again next week for another update.

If you really enjoy what we're doing here, consider becoming a paid subscriber to encourage us and gain access to the paid section, which includes insights on upcoming catalysts next week, the most shorted biotech stocks, earnings, market comments, notes and M&A comments. Now let’s dive into last week recap!

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Table of contents

  • How the market performed this week

  • Licenses / Partnerships

  • Clinical trials

  • Financing

  • RIF

  • Disease of the week

  • What I’ve read this week

Paid Content Section

  • Upcoming Catalysts Next Week

  • Reminders

  • Biotech Valuation

  • Most Shorted Biotech Stock as of This Week

  • Market

  • Notes from analysts

  • Venture Capital Market

  • M&A

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How the market performed this week

ETFs

Gene Therapy

RNA Therapy

Big Pharma

Gainers / Decliners this week in biotech

News of the week

Licenses/Partnerships

  • Clinuvel Pharmaceuticals Limited (CLVLY)

    • Event Phase: Approved

    • Drug: Scenesse

    • Indication: Porphyria

    • Target: Melanocortin (MC) receptors

    • LOA: 100%

    • Source Link

  • AbbVie Inc. (ABBV)

    • Event Phase: II

    • Drug: NX-13

    • Indication: Ulcerative Colitis (UC)

    • Target: Mitochondria

    • LOA: 19%

    • Source Link

  • AbbVie Inc. (ABBV)

    • Event Phase: I

    • Drug: NX-13

    • Indication: Crohn's Disease

    • Target: Mitochondria

    • LOA: 11%

    • Source Link

  • AbbVie Inc. (ABBV)

    • Event Phase: Preclinical

    • Drug: LABP-66

    • Indication: Multiple Sclerosis (MS)

    • Target: Mitochondria

    • Source Link

  • AbbVie Inc. (ABBV)

    • Event Phase: Preclinical

    • Drug: LABP-66

    • Indication: Alzheimer's Disease (AD)

    • Target: Mitochondria

    • Source Link

  • AbbVie Inc. (ABBV)

    • Event Phase: Preclinical

    • Drug: LABP-69

    • Indication: Rheumatoid Arthritis (RA)

    • Target: Plexin Domain Containing 2 (PLXDC2)

    • Source Link

  • AbbVie Inc. (ABBV)

    • Event Phase: Preclinical

    • Drug: LABP-73

    • Indication: Asthma

    • Target: Mitochondria

    • Source Link

  • Active Biotech AB (ACTI)

    • Event Phase: II

    • Drug: TASQ

    • Indication: Multiple Myeloma (MM)

    • Target: Angiogenesis, Migration inhibitory factor-related protein 14 (MRP14)/S100A9

    • LOA: 11%

    • Source Link

  • Acorda Therapeutics, Inc. (ACOR)

    • Event Phase: Approved

    • Drug: Inbrija

    • Indication: Parkinson's Disease (PD)

    • Target: Dopamine Receptor - Unspecified

    • LOA: 100%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Triple-Negative Breast Cancer (TNBC)

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Uterine (Endometrial) Cancer

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: Development Outside U.S.

    • Drug: PRO1102

    • Indication: Solid Tumors

    • Target: HER2/neu or ErbB-2

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: Development Outside U.S.

    • Drug: PRO1106

    • Indication: Solid Tumors

    • Target: Slitrk6

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: Development Outside U.S.

    • Drug: PRO1286

    • Indication: Solid Tumors

    • Target: EGFR (Epidermal Growth Factor Receptor)

    • Source Link

  • BeiGene, Ltd. (BGNE)

    • Event Phase: III

    • Drug: Tevimbra

    • Indication: Non-Small Cell Lung Cancer (NSCLC)

    • Target: Immune System, Programmed death-1 receptor (PD-1)

    • LOA: 44%

    • Source Link

  • Dr. Reddy's Laboratories Ltd. (RDY)

    • Event Phase: Development Outside U.S.

    • Drug: AVT03

    • Indication: Bone Complications (Including Bone Metastases)

    • Target: RANK Ligand (RANKL)

    • Source Link

  • Qurient Co., Ltd. (115180)

    • Event Phase: II

    • Drug: Q901

    • Indication: Solid Tumors

    • Target: Cyclin Dependent Kinase 7 (CDK-7)

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1184

    • Indication: Solid Tumors

    • Target: Folate Receptor (FOLR1)

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1160

    • Indication: Solid Tumors

    • Target: Cluster of Differentiation 70 (CD70)

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Bladder Cancer

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Esophageal Cancer

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Gastric Cancer

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Non-Small Cell Lung Cancer (NSCLC)

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Ovarian Cancer

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

  • Genmab A/S (GMAB)

    • Event Phase: II

    • Drug: PRO1107

    • Indication: Solid Tumors

    • Target: Tyrosine Kinases

    • LOA: 11%

    • Source Link

Clinical trials (LOA=likelihood of approval)

  1. Evaxion Biotech A/S (EVAX)

    • Drug: EVX-01

    • Indication: Melanoma

    • Target: Immune System, Tumor Cells

    • Event Phase: Phase IIb (w/Keytruda)

    • LOA: -

    • Source Link: Link

  2. Affimed N.V. (AFMD)

    • Drug: AFM24

    • Indication: Solid Tumors

    • Target: Cluster of Differentiation 16 (CD16), EGFR

    • Event Phase: Phase I/IIa (w/Atezolizumab)

    • LOA: 11%

    • Source Link: Link

  3. Bio-Path Holdings, Inc. (BPTH)

    • Drug: Prexigebersen

    • Indication: Acute Myelogenous Leukemia (AML)

    • Target: Growth factor receptor bound protein 2 (Grb-2)

    • Event Phase: Phase II (w/Decitabine)

    • LOA: 12%

    • Source Link: Link

  4. FibroGen, Inc. (FGEN)

    • Drug: FG-3246

    • Indication: Prostate Cancer

    • Target: Cluster of Differentiation 46 (CD46)

    • Event Phase: Phase Ib/II (w/ Enzalutamide)

    • LOA: 11%

    • Source Link: Link

  5. Iovance Biotherapeutics, Inc. (IOVA)

    • Drug: Amtagvi

    • Indication: Melanoma

    • Target: Immune System, Stem Cells/Other Cell Therapies, T lymphocytes

    • Event Phase: Approved (Phase II)

    • LOA: 100%

    • Source Link: Link

  6. Candel Therapeutics, Inc. (CADL)

    • Drug: CAN-2409

    • Indication: Non-Small Cell Lung Cancer (NSCLC)

    • Target: Thymidine Kinase, Tumor Cells

    • Event Phase: Phase II

    • LOA: 11%

    • Source Link: Link

  7. Alligator Bioscience AB (ATORX)

    • Drug: ADC-1013

    • Indication: Pancreatic Cancer

    • Target: Cluster of Differentiation 40 (CD40), Immune System

    • Event Phase: Phase Ib/II (EU)

    • LOA: -

    • Source Link: Link

  8. Pfizer Inc. (PFE)

    • Drug: Tivdak

    • Indication: Solid Tumors

    • Target: Tissue Factor

    • Event Phase: Phase II

    • LOA: 11%

    • Source Link: Link

  9. Genmab A/S (GMAB)

    • Drug: Epkinly

    • Indication: Follicular Lymphoma (FL)

    • Target: Cluster of Differentiation 20 (CD20), Cluster of Differentiation 3 (CD3)

    • Event Phase: NDA/BLA (Phase I/II)

    • LOA: 92%

    • Source Link: Link

  10. Novartis AG (NVS)

    • Drug: ARV-766

    • Indication: Prostate Cancer

    • Target: Androgen receptors

    • Event Phase: Phase I/II (w/Abiraterone)

    • LOA: 11%

    • Source Link: Link

  11. Johnson & Johnson (JNJ)

    • Drug: Rybrevant

    • Indication: Non-Small Cell Lung Cancer (NSCLC)

    • Target: EGFR, c-Met (HGFR)

    • Event Phase: Approved (Phase III)

    • LOA: 100%

    • Source Link: Link

  12. Novartis AG (NVS)

    • Drug: DFF332

    • Indication: Renal Cell Cancer (RCC)

    • Target: Hypoxia Inducible Factor-2 Alpha (HIF-2a)

    • Event Phase: Phase I

    • LOA: 5%

    • Source Link: Link

  13. Agenus Inc. (AGEN)

    • Drug: Botensilimab

    • Indication: Colorectal Cancer (CRC)

    • Target: Cytotoxic T-Lymphocyte Antigen 4 (CTLA4)

    • Event Phase: Phase Ia/Ib (PK/PD Study)

    • LOA: 11%

    • Source Link: Link

  14. Bristol Myers Squibb Company (BMY)

    • Drug: Onureg

    • Indication: Myelodysplastic Syndrome (MDS)

    • Target: DNA Methyltransferase (DNMT)

    • Event Phase: Phase II/III

    • LOA: 37%

    • Source Link: Link

  15. Merus N.V. (MRUS)

    • Drug: MCLA-145

    • Indication: Solid Tumors

    • Target: Cluster of Differentiation 137 (CD 137), Immune System, PD-L1

    • Event Phase: Phase I

    • LOA: 5%

    • Source Link: Link

  16. Eli Lilly and Company (LLY)

    • Drug: Verzenio

    • Indication: Prostate Cancer

    • Target: Cyclin Dependent Kinase 4 (CDK-4), Cyclin Dependent Kinase 6 (CDK-6)

    • Event Phase: Phase II/III

    • LOA: 11%

    • Source Link: Link

  17. Karyopharm Therapeutics (KPTI)

    • Drug: Xpovio

    • Indication: Peripheral T-Cell Lymphoma (PTCL) - NHL

    • Target: Exportin-1/CRM1/XPO1, Immune System, Interferon-beta (IFNb), SARS-CoV-2

    • Event Phase: Phase Ib

    • LOA: -

    • Source Link: Link

  18. AstraZeneca PLC (AZN)

    • Drug: ATG-017

    • Indication: Solid Tumors

    • Target: MAPK/ERK, MAPK1/ERK2, MAPK3/ERK1

    • Event Phase: Phase I

    • LOA: 5%

    • Source Link: Link

  19. Blueprint Medicines Corporation (BPMC)

    • Drug: BLU-222

    • Indication: HR+/HER2- Breast Cancer

    • Target: Cyclin Dependent Kinase 2 (CDK-2)

    • Event Phase: Phase I/II

    • LOA: 11%

    • Source Link: Link

  20. Coherus BioSciences, Inc.

    • Ticker: CHRS

    • Phase: II

    • Trial: Phase I/II - Dose Escalation/Expansion

    • Drug: CHS-114

    • Indication: Solid Tumors

    • Target: Chemokine Receptor 8 (CCR8)

    • Response Rate: 11%

    • Link: News Release

  21. Bristol Myers Squibb Company

    • Ticker: BMY

    • Phase: Approved

    • Trial: Phase III - TRANSFORM

    • Drug: Breyanzi

    • Indication: Diffuse Large B-Cell Lymphoma (DLBCL) - NHL

    • Target: Autologous Chimeric Antigen Receptor T-cells (CAR-T), Cluster of Differentiation 19 (CD19), Immune System, T lymphocytes

    • Response Rate: 100%

    • Link: News Release

  22. Cantargia AB

    • Ticker: CANTA

    • Phase: II

    • Trial: Phase I/IIa - CANFOUR (BeNeLux/Scandinavia), Preclinical Studies

    • Drug: CAN-04

    • Indication: Pancreatic Cancer

    • Target: IL-1 Receptor (IL-1R)

    • Response Rate: 11%

    • Link: News Release

  23. AADi Bioscience, Inc.

    • Ticker: AADI

    • Phase: Preclinical

    • Trial: Preclinical Studies

    • Drug: Fyarro

    • Indication: Non-Small Cell Lung Cancer (NSCLC)

    • Target: Mammalian Target of Rapamycin (mTOR)/mTORC

    • Link: News Release

  24. Azitra Inc.

    • Ticker: AZTR

    • Phase: Preclinical

    • Trial: Preclinical Studies

    • Drug: ATR-04

    • Indication: Staphylococcal Vaccines and Other Staphylococcus-Specific Agents (Antibacterial)

    • Target: Microbiota (flora, microbiome) excluding the digestive tract

    • Link: News Release

  25. Roche Holding AG

    • Ticker: RHHBY

    • Phase: III

    • Trial: Phase I/II - NP30179 (w/Gazyva)

    • Drug: Columvi

    • Indication: Mantle Cell Lymphoma - NHL

    • Target: Cluster of Differentiation 20 (CD20), Cluster of Differentiation 3 (CD3), Immune System

    • Response Rate: 44%

    • Link: News Release

  26. Bristol Myers Squibb Company

    • Ticker: BMY

    • Phase: II

    • Trial: Phase I - POC - w/BMC128 (Israel)

    • Drug: Opdivo

    • Indication: Solid Tumors

    • Target: Immune System, Programmed death-1 receptor (PD-1)

    • Response Rate: 11%

    • Link: News Release

  27. Supernus Pharmaceuticals, Inc.

    • Ticker: SUPN

    • Phase: II

    • Trial: Phase IIa - RENAISSANCE

    • Drug: SPN-817

    • Indication: Partial / Focal Seizures (Epilepsy)

    • Target: Cholinesterases

    • Response Rate: 12%

    • Link: News Release

  28. HOOKIPA Pharma Inc.

    • Ticker: HOOK

    • Phase: II

    • Trial: Phase I/II - HPV-Positive Cancers (HB-201/HB-202)

    • Drug: HB-200

    • Indication: Head and Neck Cancer

    • Target: Human Papillomavirus (HPV), Immune System

    • Response Rate: 12%

    • Link: News Release

  29. Cogent Biosciences, Inc.

    • Ticker: COGT

    • Phase: III

    • Trial: Phase III - PEAK

    • Drug: Bezuclastinib

    • Indication: Gastrointestinal Stromal Tumor (GIST)

    • Target: KIT/c-KIT

    • Response Rate: 45%

    • Link: News Release

  30. Kronos Bio, Inc.

    • Ticker: KRON

    • Phase: II

    • Trial: Phase I/II - MYC-Amplified

    • Drug: KB-0742

    • Indication: Solid Tumors

    • Target: Cyclin Dependent Kinase 9 (CDK-9)

    • Response Rate: 11%

    • Link: News Release

  31. Compass Therapeutics Inc

    • Ticker: CMPX

    • Phase: I

    • Trial: Phase Ib - CTX-471-001

    • Drug: CTX-471

    • Indication: Solid Tumors

    • Target: Cluster of Differentiation 137 (CD 137), Immune System

    • Response Rate: 11%

    • Link: News Release

  32. Amgen Inc.

    • Ticker: AMGN

    • Phase: III

    • Trial: Phase III - THOR

    • Drug: Lumakras

    • Indication: Non-Small Cell Lung Cancer (NSCLC)

    • Target: Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS)

    • Link: News Release

Financing events

  1. AltruBio

    • Description: Develops antibody therapeutics for cancer and immune-related diseases, focusing on antibody-drug conjugates and targeted tumor indications.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 21, 2024

    • Deal Type: Later Stage VC

    • Deal Synopsis: Raised $225 million in Series B funding led by BVF Partners to support Phase 2a and 2b clinical trials of ALTB-268 in ulcerative colitis.

    • Investors: aMoon Fund, Blackstone, BVF Partners, Cormorant Asset Management, Delos Capital Partners, RA Capital Management, Soleus Capital

    • Deal Size: $225 million

  2. Amplexd Therapeutics

    • Description: Provides early cervical cancer therapeutics, offering non-invasive treatments as alternatives to surgeries for dysplasia.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 19, 2024

    • Deal Type: Early Stage VC

    • Deal Synopsis: Raised $2 million to finalize development and IND submissions for first-in-human clinical trials.

    • Investors: Undisclosed

    • Deal Size: $2 million

  3. Better Therapeutics (PINX: BTTX)

    • Description: Develops prescription digital therapeutics for cardiometabolic diseases using cognitive behavioural therapy.

    • Verticals: AI & Machine Learning, Digital Health, Life Sciences, LOHAS & Wellness, Mobile, TMT

    • Deal Date: May 22, 2024

    • Deal Type: Merger/Acquisition

    • Deal Synopsis: To be acquired by Click Therapeutics for an undisclosed amount.

    • Investors: Click Therapeutics

  4. Bicycle Therapeutics (DUS: 50BA)

    • Description: Develops a novel class of medicines called Bicycles, focusing on oncology indications.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 23, 2024

    • Deal Type: PIPE

    • Deal Synopsis: In talks to receive $555.49 million in development capital from various investors for proprietary pipeline development.

    • Investors: Deep Track Capital, EcoR1 Capital, Fairmount Partners, Forbion, Perceptive Advisors, RA Capital Management

    • Deal Size: $555.50 million

  5. Captain T Cell

    • Description: Develops enhanced T-cell therapies for solid tumors.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 22, 2024

    • Deal Type: Seed Round

    • Deal Synopsis: Raised EUR 8.5 million to advance lead program and novel allogeneic platform.

    • Investors: Brandenburg Kapital, HIL-INVENT, i&i Biotech Fund

    • Deal Size: $9.16 million

  6. CUTISS

    • Description: Develops bio-engineered skin grafts for large skin defects.

    • Verticals: HealthTech, Life Sciences

    • Deal Date: May 21, 2024

    • Deal Type: Later Stage VC

    • Deal Synopsis: Raised CHF 25.31 million to develop personalized skin therapy denovoSkin.

    • Investors: Cherry Bay Capital Group, Giuliani

    • Deal Size: $27.83 million

  7. Dyne Therapeutics (NAS: DYN)

    • Description: Focuses on therapeutics for genetically driven muscle diseases.

    • Verticals: HealthTech, Life Sciences

    • Deal Date: May 22, 2024

    • Deal Type: Public Investment 2nd Offering

    • Deal Synopsis: Raised $325.5 million through public offering on Nasdaq.

    • Investors: Public Offering

    • Deal Size: $325.50 million

  8. En Carta Diagnostics

    • Description: Develops molecular diagnostics platform using synthetic biology.

    • Verticals: HealthTech, Life Sciences

    • Deal Date: May 22, 2024

    • Deal Type: Early Stage VC

    • Deal Synopsis: Raised EUR 1.5 million to develop a Lyme disease diagnostic kit.

    • Investors: CentraleSupélec, CVS Health

    • Deal Size: $1.62 million

  9. Enrich Biosystems

    • Description: Develops single-cell isolation technology for biomedical research.

    • Verticals: HealthTech, Life Sciences, LOHAS & Wellness

    • Deal Date: May 20, 2024

    • Deal Type: Later Stage VC

    • Deal Synopsis: Raised $2.99 million, including $750,000 in convertible debt.

    • Investors: Undisclosed

    • Deal Size: $2.99 million

  10. Fiberwood

    • Description: Manufactures sustainable wood-based thermal insulation boards.

    • Verticals: Life Sciences, LOHAS & Wellness, Manufacturing

    • Deal Date: May 23, 2024

    • Deal Type: Later Stage VC

    • Deal Synopsis: Raised EUR 7.7 million for ecological insulation and packaging development.

    • Investors: Metsä Spring, Stephen Industries

    • Deal Size: $8.29 million

  11. Foghorn Therapeutics (NAS: FHTX)

    • Description: Develops medicines targeting the chromatin regulatory system for cancer treatment.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 20, 2024

    • Deal Type: PIPE

    • Deal Synopsis: In talks to receive $70.21 million for continued development of its pipeline.

    • Investors: Undisclosed

    • Deal Size: $70.21 million

  12. Grey Wolf Therapeutics

    • Description: Develops oncology therapies focusing on immune-oncology by altering tumor cell antigens.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 23, 2024

    • Deal Type: Later Stage VC

    • Deal Synopsis: Raised GBP 99 million to develop immuno-oncology approaches.

    • Investors: Pfizer Ventures, Intermediate Capital Group, Earlybird Venture Capital, Andera Partners, British Patient Capital, Canaan Partners, Oxford Science Enterprises

    • Deal Size: $124.30 million

  13. GT Biopharma (NAS: GTBP)

    • Description: Focuses on immuno-oncology products for cancer treatment using TriKE platforms.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 23, 2024

    • Deal Type: PIPE

    • Deal Synopsis: Received $3.22 million for general corporate purposes.

    • Investors: Undisclosed

    • Deal Size: $3.22 million

  14. HI-Bio

    • Description: Develops precision medicines for autoimmune and inflammatory diseases.

    • Verticals: Life Sciences

    • Deal Date: May 22, 2024

    • Deal Type: Merger/Acquisition

    • Deal Synopsis: To be acquired by Biogen for $1.15 billion.

    • Investors: Biogen

    • Deal Size: $1.15 billion

  15. InBrain Pharma

    • Description: Develops brain infusion technology for neurodegenerative disease treatment.

    • Verticals: HealthTech, Life Sciences, TMT

    • Deal Date: May 22, 2024

    • Deal Type: Seed Round

    • Deal Synopsis: Raised EUR 3.2 million from undisclosed investors.

    • Investors: Undisclosed

    • Deal Size: $3.45 million

  16. InDex Pharmaceuticals (STO: INDEX)

    • Description: Focuses on treatments for immunological diseases, including ulcerative colitis.

    • Verticals: Life Sciences, Oncology

    • Deal Date: May 20, 2024

    • Deal Type: Merger/Acquisition

    • Deal Synopsis: To be acquired by Flerie through a reverse merger.

    • Investors: Flerie

Reduction in force (RIF)

May 21 - Lyra Therapeutics: The biotech is laying off 87 employees, including Chief Technology Officer John Bishop, Ph.D. The layoffs are effective today for 80 people and on June 20 for the remaining seven. Story

May 21 - Exscientia: The AI drug hunter is initiating “efficiency measures” to save cash, which includes a workforce reduction expected to impact between 20% and 25% of staff by the end of this year. Story  

May 20 - BIO: The biotech advocacy organization is implementing a "strategic plan" that includes laying off 30 people, according to an emailed statement from President and CEO John Crowley. The restructure is designed to "bring stronger focus and greater impact in everything we do," Crowley said. 

Disease of the week

Von Hippel-Lindau (VHL) disease is a rare genetic disorder characterized by the development of tumors and cysts in various parts of the body. It's caused by mutations in the VHL gene, which plays a crucial role in regulating cell growth and division. This gene is located on the short arm of chromosome 3.

Here's a breakdown of key aspects of Von Hippel-Lindau disease:

  1. Inheritance: VHL disease follows an autosomal dominant pattern of inheritance, meaning that a person only needs to inherit one mutated copy of the VHL gene from either parent to develop the disorder. If a parent has VHL, each of their children has a 50% chance of inheriting the mutated gene.

  2. Clinical Features: The clinical manifestations of VHL disease can vary widely among affected individuals, even within the same family. The most common features include:

    • Hemangioblastomas: These are benign tumors that often occur in the brain, spinal cord, and retina. They can cause symptoms such as headaches, vision problems, and neurological deficits.

    • Renal cell carcinoma (RCC): Individuals with VHL have an increased risk of developing kidney cancer, particularly clear cell renal cell carcinoma.

    • Pheochromocytomas and paragangliomas: These tumors arise from specialized cells of the adrenal glands (pheochromocytomas) or from nerve cells associated with the sympathetic nervous system (paragangliomas). They can cause symptoms related to excess catecholamine production, such as high blood pressure, palpitations, and sweating.

    • Pancreatic neuroendocrine tumors (PNETs): These tumors can develop in the pancreas and may produce hormones that cause various symptoms, depending on the type of hormone produced.

    • Cysts and tumors in other organs: VHL disease can also lead to the formation of cysts and tumors in the kidneys, pancreas, adrenal glands, epididymis (in males), and other organs.

  3. Diagnosis: The diagnosis of VHL disease is based on clinical features, family history, and genetic testing to identify mutations in the VHL gene. Imaging studies such as MRI or CT scans are often used to detect and monitor tumors and cysts associated with the condition.

  4. Management and Treatment: There is currently no cure for VHL disease, but treatment focuses on managing symptoms and reducing the risk of complications. This may include surveillance through regular imaging studies to monitor tumor growth, surgical removal of tumors or cysts that cause symptoms or complications, and targeted therapies for certain types of tumors, such as RCC.

  5. Genetic Counseling: Genetic counseling is recommended for individuals with VHL disease and their family members to understand the inheritance pattern, the risk of passing the condition to future generations, and the options for genetic testing and family planning.

What I’ve read this week
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