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Orchard Therapeutics hires ex-PTC executive Mark Rothera as new CEO

A 2016 Fierce 15 winner and ultrarare disease startup Orchard Therapeutics has taken on Mark Rothera as its new president and chief.

Rothera, a near 30-year biopharma vet, comes to Orchard from controversial biotech PTC Therapeutics, where he served as its chief commercial officer. He’s also done stints as global president of Aegerion Pharmaceuticals, as well as VP and GM of commercial ops at Shire Human Genetic Therapies for the EMEA region.

Orchard is focused on ex vivo autologous hematopoietic stem cell gene therapy designed to restore normal gene function in primary immune deficiencies, metabolic diseases and hematological disorders.

This tech uses a sample of the patient’s own stem cells, which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient’s body. Using the patient’s own cells (autologous) removes the need to search for a matching stem cell donor, which can take months or even years.

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PlasmaGen BioSciences raises $25 mn from Eight Roads Ventures, F-Prime

Mumbai | Biopharmaceutical company PlasmaGen BioSciences announced an investment of $25 million (Rs 160 crore) led by Eight Roads Ventures and US-based F-Prime Capital Partners.
PlasmaGeb BioSciences, which specialises in blood plasma derived products, is founded by serial entrepreneur Vinod Nahar, who also founded RSM Pharma and Trigenesis Lifesciences.
The company’s chief executive officer Ranjeet S Ajmani said: “There is a large gap between demand and supply of plasma products, not only in India but across Asia. India is strategically placed as an emerging economy with a huge demand potential.”

Eight Roads Venture is the investment arm of Fidelity International that has earlier invested in Chai Point, Medwell Ventures and Unbxd. As part of the investment, Dr Prem Pavoor, a partner at and Dr Robert Weisskoff, a partner at Capital Partners will join the board at

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Orchard Therapeutics Announces That OTL-101 Has Received a Rare Paediatric Disease Designation

London, UK — Orchard Therapeutics Limited (“Orchard”), a clinical-stage biotechnology company dedicated to bringing transformative autologous ex-vivo gene therapies to patients with rare diseases of high unmet medical need is delighted to announce today that the US Food and Drug Administration (FDA) granted a Rare Paediatric Disease Designation to OTL-101, its lead programme for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease. OTL-101 is developed in collaboration with the University of California, Los Angeles (“UCLA”) and University College London / Great Ormond Street Hospital (“UCL” and “GOSH”).

ADA-SCID is a rare inherited disorder of the immune system. ADA-SCID is caused by mutations in the gene encoding for the enzyme adenosine deaminase, which result in a severe deficiency in white blood cells and life-threatening infections. In the absence of treatment, ADA-SCID is fatal within the first year of life.

To be granted Rare Paediatric Disease Designation, a drug must be designed for the treatment of a serious or life-threatening disease which affects less than 200,000 patients in the United States and which primarily includes patients aged between 0 and 18 years.

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Menlo Therapeutics Raises $50M in Series C Financing

MENLO PARK, Calif. — Menlo Therapeutics Inc. announced today that the company has raised $50M to advance development of serlopitant, a novel NK-1 receptor antagonist, as a once-daily oral treatment for pruritus (itch) associated with atopic dermatitis, psoriasis and prurigo nodularis and for treatment of refractory chronic cough.  venBio led the financing and was joined by Novo Holdings A/S, Rock Springs Capital, Aisling Capital, and Bay City Capital as new Menlo Therapeutics investors in this Series C financing. All of Menlo Therapeutics’ Series A and B investors also participated in the financing, including Vivo Capital, Presidio Partners, Remeditex, and F-Prime Capital.

PatientPing partners with ACO in Houston

PatientPing, a health technology company that connects providers to seamlessly coordinate patient care, has partnered with Houston Methodist Coordinated Care, an Accountable Care Organization affiliated with Houston Methodist, the company said.

This partnership means that providers throughout Houston and its surrounding communities, as well as across states, will be notified when their patients are admitted, discharged or transferred to/from all eight Houston Methodist hospitals, allowing for timely intervention and overall higher quality care for patients.

PatientPing´s network includes tens of thousands of providers nationwide. In December 2016, PatientPing announced USD 31.6 million funding led by Andreessen Horowitz. PatientPing is rapidly accelerating its nationwide expansion so that any providers who share patients anywhere in the country can coordinate with one another.

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Caribou Biosciences Appoints Steven B. Kanner, Ph.D. as Chief Scientific Officer

BERKELEY, Calif.–Caribou Biosciences, Inc., a leading genome engineering company, announced today that Steven B. Kanner, Ph.D. has joined the Company as Chief Scientific Officer. Dr. Kanner brings more than 25 years of experience in drug discovery and development and will lead Caribou’s research and development of new applications for its gene editing technology.

“We are delighted to welcome such a well-respected and talented scientist as Steve to our team,” said Rachel Haurwitz, Ph.D., President and Chief Executive Officer of Caribou. “Caribou is continuing to make exciting progress in our gene editing technology development and Steve’s wealth of experience will help further accelerate our efforts.”

During his career, Dr. Kanner has held a number of research leadership roles at prominent life sciences organizations, including Bristol-Myers Squibb, Astex Pharmaceuticals, and Agensys/Astellas Pharma. Most recently, Dr. Kanner served as Vice President of Discovery Biology at Arrowhead Pharmaceuticals, where he helped lead the discovery and development of novel RNAi-based therapeutics for oncology and genetic disease programs. During his time at Bristol-Myers Squibb, he facilitated a discovery program ultimately leading to the development and launch of Sprycel® (dasatinib), a treatment for chronic myeloid leukemia.

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US HealthVest Opens Smokey Point Behavioral Hospital

MARYSVILLE, Wash.— US HealthVest announced the opening of Smokey Point Behavioral Hospital, a 115-bed psychiatric hospital that provides a full continuum of inpatient and outpatient psychiatric care to patients of all ages.

PatientPing announced a partnership with Georgia-based WellStar Health System

PatientPing announced a partnership with Georgia-based WellStar Health System.

WellStar is the largest health system in the state. It includes three skilled nursing facilities and three inpatient hospices. Providers across the state can be notified when their patients are admitted, discharged or transferred to/from a WellStar facility, the company said. By joining PatientPing, they will receive real-time notifications and care instructions, such as contact information for the patient’s care team.

“We look forward to creating an enormous, positive change across the entire patient care continuum in Georgia and beyond,” said Jay Desai, co-founder & CEO, PatientPing. “PatientPing is establishing the nation’s largest community of providers working together to coordinate patient care and reduce healthcare costs through real-time data sharing, and it’s an honor to launch Georgia with the wildly innovative team at WellStar.”

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Genoa Pharmaceuticals Secures $62 Million Series A Financing

Seattle, WA – Genoa Pharmaceuticals, Inc., a biopharmaceutical company focused on development of improved therapies for idiopathic pulmonary fibrosis (IPF) and other severe pulmonary indications, today announced the completion of a $62 million Series A financing. The round was led by F-Prime Capital Partners and Edmond de Rothschild Investment Partners, with participation by Novo AS, RiverVest Venture Partners, and TPG Biotech. Concurrent with the financing, venture capitalists Ketan Patel, M.D., Naveed Siddiqi M.D., Tiba Aynechi Ph.D., Niall O’Donnell, Ph.D., and Heather Preston, M.D. joined the board of directors. Jonathan Leff, M.D, former Executive Vice President of Research and Development at InterMune and who led the oral pirfenidone (Esbriet®) approval process, was appointed an independent director.

A. Bruce Montgomery, M.D. was appointed Chief Executive Officer and will lead clinical development out of the Seattle office. Founder and inventor, Mark Surber, Ph.D. was responsible for organizing the financing and was named Chief Scientific Officer. Dr. Surber will head the San Diego office focusing on Aerodone™ nonclinical support and pipeline research and development.

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Acacia Pharma announces positive results from its fourth and final pivotal trial of Baremsis™ for the rescue treatment of PONV

Cambridge, UK – Acacia Pharma Group Ltd (“Acacia Pharma”), the supportive care company developing products for US and international markets, announces positive results from its fourth and final pivotal Phase 3 study investigating BAREMSIS™ (amisulpride injection, formerly APD421) for the rescue treatment of patients who develop post-operative nausea & vomiting (PONV), despite having received prior antiemetic prophylaxis.

Dr Julian Gilbert, Acacia Pharma’s CEO commented: “We are delighted with these results demonstrating that BAREMSIS is safe and effective at rescuing patients who suffer PONV despite having received prior prophylaxis with standard anti-emetics. No other anti-emetic has a specific label for treating this significant unmet need and we intend to position BAREMSIS as the drug of choice for treating the 30-40% of surgical patients who suffer PONV despite prior prophylaxis, as well as for combination prophylaxis in high-risk patients.”

Acacia Pharma has now completed four pivotal Phase 3 studies of BAREMSIS successfully, all meeting their primary endpoint, and these will form the basis of the efficacy and safety package which the Company aims to submit to the US FDA as part of its New Drug Application (NDA) in 1H 2017. The Company will seek a broad and unique approval for BAREMSIS for the rescue treatment and prophylaxis of PONV, alone and in combination.

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